DK1178999T3 - L-RIBO-LNA-analoger - Google Patents

L-RIBO-LNA-analoger

Info

Publication number
DK1178999T3
DK1178999T3 DK00925080T DK00925080T DK1178999T3 DK 1178999 T3 DK1178999 T3 DK 1178999T3 DK 00925080 T DK00925080 T DK 00925080T DK 00925080 T DK00925080 T DK 00925080T DK 1178999 T3 DK1178999 T3 DK 1178999T3
Authority
DK
Denmark
Prior art keywords
ribo
compounds
lna analogues
lna
analogues
Prior art date
Application number
DK00925080T
Other languages
English (en)
Inventor
Jesper Wengel
Original Assignee
Santaris Pharma As
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=27220811&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK1178999(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Santaris Pharma As filed Critical Santaris Pharma As
Application granted granted Critical
Publication of DK1178999T3 publication Critical patent/DK1178999T3/da

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H9/00Compounds containing a hetero ring sharing at least two hetero atoms with a saccharide radical
    • C07H9/02Compounds containing a hetero ring sharing at least two hetero atoms with a saccharide radical the hetero ring containing only oxygen as ring hetero atoms
    • C07H9/04Cyclic acetals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DK00925080T 1999-05-04 2000-05-04 L-RIBO-LNA-analoger DK1178999T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
DKPA199900603 1999-05-04
DKPA199901225 1999-09-01
DKPA200000032 2000-01-11
PCT/DK2000/000225 WO2000066604A2 (en) 1999-05-04 2000-05-04 L-ribo-lna analogues

Publications (1)

Publication Number Publication Date
DK1178999T3 true DK1178999T3 (da) 2007-08-06

Family

ID=27220811

Family Applications (1)

Application Number Title Priority Date Filing Date
DK00925080T DK1178999T3 (da) 1999-05-04 2000-05-04 L-RIBO-LNA-analoger

Country Status (16)

Country Link
US (1) US7053207B2 (da)
EP (1) EP1178999B1 (da)
JP (1) JP2002543214A (da)
KR (1) KR100782896B1 (da)
CN (2) CN102180924A (da)
AT (1) ATE356824T1 (da)
AU (1) AU776362B2 (da)
CA (1) CA2372085C (da)
DE (1) DE60033927T2 (da)
DK (1) DK1178999T3 (da)
ES (1) ES2283298T3 (da)
HK (1) HK1048322A1 (da)
IL (1) IL145497A0 (da)
NZ (1) NZ514348A (da)
PT (1) PT1178999E (da)
WO (1) WO2000066604A2 (da)

Families Citing this family (894)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7119184B2 (en) * 1991-08-12 2006-10-10 Isis Pharmaceuticals, Inc. Oligonucleotides having A-DNA form and B-DNA form conformational geometry
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) * 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
TR200102328T2 (tr) * 1999-02-12 2002-01-21 Sankyo Company Limited Yeni nükleosid ve oligonükleotid analogları
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
DK1163250T3 (da) * 1999-03-24 2006-11-13 Exiqon As Forbedret syntese af [2.2.1]bicyclonukleosider
CA2395320A1 (en) * 1999-12-23 2001-07-05 Exiqon A/S Therapeutic uses of lna-modified oligonucleotides
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US6936467B2 (en) 2000-03-27 2005-08-30 University Of Delaware Targeted chromosomal genomic alterations with modified single stranded oligonucleotides
AU4948801A (en) 2000-03-27 2001-10-08 Univ Delaware Targeted chromosomal genomic alterations with modified single stranded oligonucleotides
AU2002303176A1 (en) 2001-03-27 2002-10-15 University Of Delaware Genomics applications for modified oligonucleotides
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
GB0114719D0 (en) * 2001-06-15 2001-08-08 Glaxo Group Ltd Compound
US9261460B2 (en) 2002-03-12 2016-02-16 Enzo Life Sciences, Inc. Real-time nucleic acid detection processes and compositions
JP2005514005A (ja) 2001-09-04 2005-05-19 エクシコン エ/エス 新規のlna組成物およびその使用
US7468244B2 (en) 2001-09-27 2008-12-23 University Of Delaware Polymorphism detection and separation
US7112405B2 (en) 2001-09-27 2006-09-26 University Of Delaware Compositions and methods for enhancing oligonucleotide-mediated gene alteration
AU2002351077A1 (en) * 2001-11-05 2003-05-19 Exiqon A/S Oligonucleotides modified with novel alpha-l-rna analogues
WO2003059929A1 (en) * 2001-12-21 2003-07-24 Applera Corporation Heteroconfigurational polynucleotide and methods of use
EP1572902B1 (en) 2002-02-01 2014-06-11 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
DE60329277D1 (de) 2002-02-01 2009-10-29 Life Technologies Corp Oligonukleotidzusammensetzungen mit verbesserter effizienz
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US7566535B2 (en) 2002-03-07 2009-07-28 University Of Delaware Enhanced oligonucleotide-mediated nucleic acid sequence alteration
US9353405B2 (en) 2002-03-12 2016-05-31 Enzo Life Sciences, Inc. Optimized real time nucleic acid detection processes
EP1495139A4 (en) * 2002-03-29 2006-06-28 Genentech Inc METHODS AND COMPOSITIONS FOR DETECTING AND QUANTIFYING NUCLEIC ACID ANALYTES
DK2264172T3 (da) 2002-04-05 2017-11-27 Roche Innovation Ct Copenhagen As Oligomerforbindelser til modulering af HIF-1á-ekspression
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
CA2484526C (en) * 2002-05-08 2009-10-13 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003243541A1 (en) * 2002-06-12 2003-12-31 Ambion, Inc. Methods and compositions relating to labeled rna molecules that reduce gene expression
US20100075423A1 (en) * 2002-06-12 2010-03-25 Life Technologies Corporation Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference
US20040248094A1 (en) * 2002-06-12 2004-12-09 Ford Lance P. Methods and compositions relating to labeled RNA molecules that reduce gene expression
US7005265B1 (en) 2002-06-20 2006-02-28 Wenhong Fan Nonenzymatic catalytic signal amplification for nucleic acid hybridization assays
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
DK2284269T3 (da) * 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense-design
AU2013201786B2 (en) * 2002-11-18 2015-04-02 Roche Innovation Center Copenhagen A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
EP1426448A1 (en) * 2002-12-03 2004-06-09 bioMérieux BV Method for lowering the effects of sequence variations in a diagnostic hybridization assay, probe for use in the assay and assay
US20060292438A1 (en) * 2002-12-23 2006-12-28 Applera Corporation; Applied Biosystems Group Heteroconfigurational Polynucleotides and Methods of Use
US7713738B2 (en) 2003-02-10 2010-05-11 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
EP1592793B2 (en) 2003-02-10 2014-05-07 Santaris Pharma A/S Oligomeric compounds for the modulation of survivin expression
WO2004083430A2 (en) 2003-03-21 2004-09-30 Santaris Pharma A/S SHORT INTERFERING RNA (siRNA) ANALOGUES
WO2004106356A1 (en) * 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
ATE553216T1 (de) 2003-06-20 2012-04-15 Exiqon As Sonden, bibliotheken und kits zur analyse von nukleinsäuregemischen und verfahren zu deren konstruktion
US7683036B2 (en) * 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US7480382B2 (en) * 2003-09-30 2009-01-20 Microsoft Corporation Image file container
EP1688493B1 (en) * 2003-11-07 2008-08-13 Daiichi Sankyo Company, Limited Method of detecting genetic polymorphism
RU2377301C2 (ru) 2003-12-23 2009-12-27 Сантарис Фарма А/С ОЛИГОМЕРНОЕ СОЕДИНЕНИЕ, ПОНИЖАЮЩЕЕ ЭКСПРЕССИЮ ЧЕЛОВЕЧЕСКОГО ГЕНА Bcl-2, КОНЪЮГАТ, ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ И ПРИМЕНЕНИЕ ОЛИГОМЕРНОГО СОЕДИНЕНИЯ ДЛЯ ЛЕЧЕНИЯ РАКА
GB0406864D0 (en) 2004-03-26 2004-04-28 Qiagen As Nucleic acid sequencing
US8192937B2 (en) 2004-04-07 2012-06-05 Exiqon A/S Methods for quantification of microRNAs and small interfering RNAs
CA2557532A1 (en) 2004-04-23 2005-11-10 Angela M. Christiano Inhibition of hairless protein mrna
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
JP5435864B2 (ja) 2004-05-28 2014-03-05 アシュラジェン インコーポレイテッド マイクロrnaに関与する方法および組成物
US9447138B2 (en) 2004-11-09 2016-09-20 Roche Innovation Center Copenhagen A/S Potent LNA oligonucleotides for the inhibition of HIF-1a expression
WO2006050734A2 (en) 2004-11-09 2006-05-18 Santaris Pharma A/S Potent lna oligonucleotides for the inhibition of hif-1a expression
DE602005023772D1 (de) * 2004-11-09 2010-11-04 Santaris Pharma As Lna-oligonukleotide und krebsbehandlung
MX2007005557A (es) 2004-11-09 2008-02-15 Santaris Pharma As Oligonucleotidos lna y el tratamiento de cancer.
EP2322616A1 (en) 2004-11-12 2011-05-18 Asuragen, Inc. Methods and compositions involving miRNA and miRNA inhibitor molecules
US20060166238A1 (en) * 2004-12-22 2006-07-27 Ramsing Niels B Probes, libraries and kits for analysis of mixtures of nucleic acids and methods for constructing the same
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
EP1838870A2 (en) 2004-12-29 2007-10-03 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
EP2360249A1 (en) 2005-03-31 2011-08-24 Calando Pharmaceuticals, Inc. Inhibitors of ribonucleotide reductase subunit 2 and uses thereof
US7838502B2 (en) 2005-05-06 2010-11-23 University Of Massachusetts Medical School Compositions and methods to modulate H. influenzae pathogenesis
ES2702531T3 (es) * 2005-06-23 2019-03-01 Biogen Ma Inc Composiciones y procedimientos para modulación de splicing de SMN2
CA2617693A1 (en) 2005-08-17 2007-02-22 Medexis S.A. Composition and method for determination of ck19 expression
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
JP5523705B2 (ja) 2005-08-29 2014-06-18 レグルス・セラピューティクス・インコーポレイテッド Mir−122aをモジュレートする使用方法
EP1971371B1 (en) 2005-12-01 2015-08-05 Pronai Therapeutics, Inc. Cancer therapies and pharmaceutical compositions used therein
WO2007073149A1 (en) 2005-12-22 2007-06-28 Keygene N.V. Alternative nucleotides for improved targeted nucleotide exchange
WO2007087113A2 (en) 2005-12-28 2007-08-02 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
CN102908630B (zh) 2006-01-27 2014-11-19 Isis制药公司 6-修饰的双环核酸类似物
CA2640058C (en) 2006-01-27 2018-04-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
JP5829373B2 (ja) 2006-01-27 2015-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. Nogoレセプターアンタゴニスト
DK2002004T3 (da) 2006-03-23 2015-11-30 Roche Innovation Ct Copenhagen As Lille internt segmenteret interferens-rna
EA201100811A1 (ru) 2006-04-03 2012-06-29 Сантарис Фарма А/С Фармацевтическая композиция
WO2007112753A2 (en) 2006-04-03 2007-10-11 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
WO2007136988A2 (en) * 2006-05-05 2007-11-29 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of gccr
EP2527442A3 (en) 2006-05-05 2013-03-06 Isis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US7666854B2 (en) * 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
EP2054415A1 (en) * 2006-07-14 2009-05-06 Santaris Pharma A/S Adenosine receptor antagonists
EP2410053B2 (en) 2006-10-18 2020-07-15 Ionis Pharmaceuticals, Inc. Antisense compounds
EP2090665A2 (en) 2006-10-20 2009-08-19 Exiqon A/S Novel human microRNAs associated with cancer
US8188255B2 (en) 2006-10-20 2012-05-29 Exiqon A/S Human microRNAs associated with cancer
US20100062436A1 (en) 2006-10-31 2010-03-11 Noxxon Pharma Ag Methods for Detection of a Single- or Double-Stranded Nucleic Acid Molecule
US8420613B2 (en) 2006-11-15 2013-04-16 The J. David Gladstone Institutes Methods and compositions for reducing amyloid beta levels
TW200838551A (en) * 2006-11-27 2008-10-01 Isis Pharmaceuticals Inc Methods for treating hypercholesterolemia
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP3536788A1 (en) 2006-12-21 2019-09-11 QIAGEN GmbH Microrna target site blocking oligos and uses thereof
WO2008086807A2 (en) 2007-01-19 2008-07-24 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
US20080206850A1 (en) * 2007-02-28 2008-08-28 Dellinger Douglas J Methods and compositions for DNA synthesis
WO2008113830A1 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
US8999634B2 (en) 2007-04-27 2015-04-07 Quest Diagnostics Investments Incorporated Nucleic acid detection combining amplification with fragmentation
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (da) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocycliske bicycliske nukleinsyreanaloge
WO2008151631A2 (en) * 2007-06-15 2008-12-18 Exiqon A/S Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis
EP2176280B2 (en) * 2007-07-05 2015-06-24 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US8361714B2 (en) 2007-09-14 2013-01-29 Asuragen, Inc. Micrornas differentially expressed in cervical cancer and uses thereof
DK2548962T3 (da) 2007-09-19 2016-04-11 Applied Biosystems Llc Sirna-sekvensuafhængige modifikationsformater til reduktion af off-target-fænotypeeffekter i RNAI og stabiliserede former deraf
MX2010003465A (es) 2007-10-02 2010-07-05 Amgen Inc Incremento de eritropoyetina utilizando acidos nucleicos que hibridan para micro-arn y precursores de los mismos.
ES2689508T3 (es) 2007-10-04 2018-11-14 Roche Innovation Center Copenhagen A/S Micromir
WO2009067647A1 (en) * 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP3561494B1 (en) 2007-12-06 2022-03-02 Genalyte, Inc. METHOD FOR IDENTIFYING A SEQUENCE OF NUCLEOTIDES IN AN UNKNOWN SPECIES OF NUCLEIC ACID 
AND DEVICE FOR CARRYING OUT THE METHOD
CA2708751A1 (en) 2007-12-14 2009-06-25 Minitube Of America, Inc. Gender-specific separation of sperm cells and embryos
WO2009100320A2 (en) * 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
US8361980B2 (en) 2008-03-07 2013-01-29 Santaris Pharma A/S Pharmaceutical compositions for treatment of microRNA related diseases
US8227431B2 (en) 2008-03-17 2012-07-24 Hetero Drugs Limited Nucleoside derivatives
WO2009124295A2 (en) * 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
DK2285819T3 (da) * 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
WO2009123764A2 (en) 2008-04-04 2009-10-08 Calando Pharmaceuticals, Inc. Compositions and use of epas1 inhibitors
US8258111B2 (en) 2008-05-08 2012-09-04 The Johns Hopkins University Compositions and methods related to miRNA modulation of neovascularization or angiogenesis
US8222221B2 (en) 2008-06-04 2012-07-17 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
CA2635187A1 (en) * 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
WO2010014592A1 (en) 2008-07-29 2010-02-04 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
EP2315832B1 (en) 2008-08-01 2015-04-08 Roche Innovation Center Copenhagen A/S Micro-rna mediated modulation of colony stimulating factors
US20110237646A1 (en) * 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
EP2356129B1 (en) * 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
CA2739464C (en) * 2008-10-03 2020-03-31 Opko Curna, Llc Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
RU2535964C2 (ru) 2008-10-15 2014-12-20 АйЭсАйЭс ФАРМАСЬЮТИКАЛЗ, ИНК. Модуляция экспрессии фактора 11
EP2358398A2 (en) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
ES2939310T3 (es) 2008-10-27 2023-04-20 Genalyte Inc Biosensores basados en sondeo y detección ópticos
KR101866152B1 (ko) 2008-12-04 2018-06-08 큐알엔에이, 인크. 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
WO2010065671A2 (en) 2008-12-04 2010-06-10 Curna, Inc. Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
CN102317458B (zh) 2008-12-04 2018-01-02 库尔纳公司 通过红细胞生成素(epo)天然反义转录物的抑制对epo相关疾病的治疗
EP2376637A1 (en) 2008-12-22 2011-10-19 Keygene N.V. Use of double stranded rna to increase the efficiency of targeted gene alteration in plant protoplasts
US20120021515A1 (en) 2009-02-06 2012-01-26 Swayze Eric E Oligomeric compounds and methods
EP3009150B1 (en) 2009-02-12 2019-11-13 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
US9464287B2 (en) 2009-03-16 2016-10-11 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (NRF2) related diseases by inhibition of natural antisense transcript to NRF2
EP2408920B1 (en) 2009-03-17 2017-03-08 CuRNA, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
JP5773535B2 (ja) 2009-04-24 2015-09-02 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブRoche Innovation Center Copenhagen A/S インターフェロンに非応答性のhcv患者の治療のための医薬組成物
WO2010124231A2 (en) 2009-04-24 2010-10-28 The Board Of Regents Of The University Of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions
CA2761142C (en) 2009-05-06 2021-06-08 Opko Curna, Llc Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
EP2427553A4 (en) 2009-05-06 2012-11-07 Opko Curna Llc TREATMENT OF LIPID TRANSPORT AND METABOLISM-RELATED DISEASES BY INHIBITING THE NATURAL ANTISENSE TRANSCRIPT AGAINST A LIPID TRANSPORT AND METABOLIC TREATMENT
CA3185821A1 (en) 2009-05-08 2010-11-11 Curna, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
NO2432881T3 (da) 2009-05-18 2018-04-14
US8895527B2 (en) 2009-05-22 2014-11-25 Curna, Inc. Treatment of transcription factor E3 (TFE3) and insulin receptor substrate 2(IRS2) related diseases by inhibition of natural antisense transcript to TFE3
ES2618576T3 (es) 2009-05-28 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico
US9200276B2 (en) 2009-06-01 2015-12-01 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent RNA interference, compositions and methods of use thereof
KR101801404B1 (ko) 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
CA2765509C (en) 2009-06-16 2021-08-17 Joseph Collard Treatment of paraoxonase 1 (pon1) related diseases by inhibition of natural antisense transcript to pon1
MX2011014004A (es) 2009-06-17 2012-06-19 Isis Pharmaceuticals Inc Composiciones y metodos para la modulacion de division de smn2 en un sujeto.
ES2618894T3 (es) 2009-06-24 2017-06-22 Curna, Inc. Tratamiento de enfermedades relacionadas con el receptor del factor de necrosis tumoral 2 (tnfr2) por inhibición del transcrito natural antisentido para tnfr2
CA2765815A1 (en) 2009-06-26 2010-12-29 Opko Curna, Llc Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
EP2456870A1 (en) 2009-07-21 2012-05-30 Santaris Pharma A/S Antisense oligomers targeting pcsk9
CA2768947C (en) 2009-07-24 2018-06-19 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
EP2462229B1 (en) 2009-08-05 2016-05-11 CuRNA, Inc. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
KR101827015B1 (ko) 2009-08-11 2018-02-07 큐알엔에이, 인크. 아디포넥틴(adipoq)에 대한 천연 안티센스 전사체의 억제에 의한 아디포넥틴(adipoq) 관련된 질환의 치료
WO2011022606A2 (en) 2009-08-21 2011-02-24 Curna, Inc. Treatment of 'c terminus of hsp70-interacting protein' (chip) related diseases by inhibition of natural antisense transcript to chip
WO2011031482A2 (en) 2009-08-25 2011-03-17 Curna, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
US20110196141A1 (en) * 2009-09-07 2011-08-11 Council Of Scientific & Industrial Research Locked and unlocked 2'-o phosphoramidite nucleosides, process of preparation thereof and oligomers comprising the nucleosides
WO2011032034A2 (en) 2009-09-10 2011-03-17 University Of Idaho Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids
ES2664591T3 (es) 2009-09-25 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con la filagrina (flg) mediante la modulación de la expresión y actividad del gen FLG
KR101860175B1 (ko) 2009-10-26 2018-05-21 에프. 호프만-라 로슈 아게 글리코실화된 면역글로불린의 제조 방법
WO2011053994A1 (en) 2009-11-02 2011-05-05 Alnylam Pharmaceuticals, Inc. Modulation of ldl receptor gene expression with double-stranded rnas targeting the ldl receptor gene promoter
EP2499248B1 (en) 2009-11-13 2017-01-04 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
EP2513310B1 (en) 2009-12-16 2017-11-01 CuRNA, Inc. Treatment of membrane bound transcription factor peptidase, site 1 (mbtps1) related diseases by inhibition of natural antisense transcript to mbtps1
KR101891352B1 (ko) 2009-12-23 2018-08-24 큐알엔에이, 인크. 간세포 성장 인자(hgf)에 대한 천연 안티센스 전사체의 억제에 의한 hgf 관련 질환의 치료
CA2782375C (en) 2009-12-23 2023-10-31 Opko Curna, Llc Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
EP2519633B1 (en) 2009-12-29 2017-10-25 CuRNA, Inc. Treatment of nuclear respiratory factor 1 (nrf1) related diseases by inhibition of natural antisense transcript to nrf1
RU2611186C2 (ru) 2009-12-29 2017-02-21 Курна, Инк. ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С ОПУХОЛЕВЫМ БЕЛКОМ 63 (р63), ПУТЕМ ИНГИБИРОВАНИЯ ПРИРОДНОГО АНТИСМЫСЛОВОГО ТРАНСКРИПТА К р63
RU2016115782A (ru) 2009-12-31 2018-11-28 Курна, Инк. Лечение заболеваний, связанных с субстратом рецептора инсулина 2 (irs2), путем ингибирования природного антисмыслового транскрипта к irs2 и транскрипционному фактору е3 (tfe3)
DK2521784T3 (da) 2010-01-04 2018-03-12 Curna Inc Behandling af interferon regulatorisk faktor 8- (irf8) relaterede sygdomme ved hæmning af naturligt antisense-transskript mod irf8
CN102822342B (zh) 2010-01-06 2017-05-10 库尔纳公司 通过抑制胰腺发育基因的天然反义转录物而治疗胰腺发育基因相关疾病
CN111700901A (zh) 2010-01-08 2020-09-25 Ionis制药公司 血管生成素样3表达的调节
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
ES2664866T3 (es) 2010-01-11 2018-04-23 Curna, Inc. Tratamiento de enfermedades relacionadas con la globulina fijadora de hormonas sexuales (shbg) mediante inhibición del transcrito antisentido natural a shbg
EP2524038A4 (en) * 2010-01-12 2013-11-20 Isis Pharmaceuticals Inc MODULATION OF TRANSFORMING A GROWTH FACTOR BETA-1 EXPRESSION
CN102782135A (zh) 2010-01-25 2012-11-14 库尔纳公司 通过抑制rna酶h1的天然反义转录物而治疗rna酶h1相关疾病
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
AU2011213562B2 (en) 2010-02-08 2016-07-07 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP3208347B1 (en) 2010-02-08 2019-08-14 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
CN102844435B (zh) 2010-02-22 2017-05-10 库尔纳公司 通过抑制吡咯啉‑5‑羧酸还原酶1(pycr1)的天然反义转录物而治疗pycr1相关疾病
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011106689A1 (en) * 2010-02-26 2011-09-01 Isis Pharmaceuticals, Inc. Modulation of smad3 expression
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
US8906875B2 (en) 2010-03-12 2014-12-09 The Brigham And Women's Hospital, Inc. Methods of treating vascular inflammatory disorders
EP2545173A2 (en) 2010-03-12 2013-01-16 Sarepta Therapeutics, Inc. Antisense modulation of nuclear hormone receptors
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
KR101877065B1 (ko) 2010-04-02 2018-07-10 큐알엔에이, 인크. 집락 자극 인자 3 (csf3)에 대한 자연 안티센스 전사체의 저해에 의한 집락 자극 인자 3 (csf3) 관련된 질환의 치료
AU2011237630B2 (en) 2010-04-06 2016-01-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of CD274/PD-L1 gene
CA2795281A1 (en) 2010-04-09 2011-10-13 Curna, Inc. Treatment of fibroblast growth factor 21 (fgf21) related diseases by inhibition of natural antisense transcript to fgf21
JP6066900B2 (ja) 2010-04-26 2017-01-25 エータイアー ファーマ, インコーポレイテッド システイニルtRNA合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
AU2011248614B2 (en) 2010-04-27 2017-02-16 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of isoleucyl tRNA synthetases
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
US9156873B2 (en) 2010-04-28 2015-10-13 Isis Pharmaceuticals, Inc. Modified 5′ diphosphate nucleosides and oligomeric compounds prepared therefrom
EP3091027B1 (en) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
EP2563911B1 (en) 2010-04-28 2021-07-21 aTyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of alanyl trna synthetases
JP5896175B2 (ja) 2010-04-29 2016-03-30 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. トランスサイレチン発現の調節
CN103118693B (zh) 2010-04-29 2017-05-03 Atyr 医药公司 与缬氨酰‑tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
EP2563912B1 (en) 2010-04-29 2018-09-05 aTyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of asparaginyl trna synthetases
US20130156845A1 (en) 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
SG184938A1 (en) 2010-04-30 2012-11-29 Exiqon As In situ hybridization method and buffer
US8981045B2 (en) 2010-05-03 2015-03-17 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of methionyl-tRNA synthetases
EP2566515B1 (en) 2010-05-03 2017-08-02 aTyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of arginyl-trna synthetases
CA2797977C (en) 2010-05-03 2019-08-20 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of phenylalanyl-alpha-trna synthetases
EP2957636B1 (en) 2010-05-03 2020-04-01 CuRNA, Inc. Treatment of sirtuin 3 (sirt3) related diseases by inhibition of natural antisense transcript to sirt3
AU2011248101B2 (en) 2010-05-04 2016-10-20 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of p38 multi-tRNA synthetase complex
US9238042B2 (en) 2010-05-13 2016-01-19 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
WO2011143482A2 (en) 2010-05-14 2011-11-17 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of phenylalanyl-beta-trna synthetases
CN103096914B (zh) 2010-05-17 2015-08-12 Atyr医药公司 与亮氨酰-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
NO2576784T3 (da) 2010-05-26 2018-04-14
CA2799207C (en) 2010-05-26 2019-03-26 Curna, Inc. Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1
JP6046607B2 (ja) 2010-05-27 2016-12-21 エータイアー ファーマ, インコーポレイテッド グルタミニルtRNA合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
AU2011261434B2 (en) 2010-06-02 2015-11-26 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011156278A1 (en) 2010-06-07 2011-12-15 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US9518259B2 (en) 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
ES2863526T3 (es) 2010-06-23 2021-10-11 Curna Inc Tratamiento de enfermedades relacionadas con la subunidad alfa del canal de sodio (SCNA), controlado por voltaje, mediante inhibición de la transcripción antisentido natural a SCNA
WO2012021247A2 (en) 2010-07-12 2012-02-16 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glycyl-trna synthetases
WO2012009402A2 (en) 2010-07-14 2012-01-19 Opko Curna Llc Treatment of discs large homolog (dlg) related diseases by inhibition of natural antisense transcript to dlg
PL3031920T3 (pl) 2010-07-19 2020-01-31 Ionis Pharmaceuticals, Inc. Modulowanie ekspresji kinazy białkowej dystrofii miotonicznej (dmpk)
WO2012027206A1 (en) 2010-08-24 2012-03-01 Merck Sharp & Dohme Corp. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
CN103108650A (zh) 2010-08-25 2013-05-15 Atyr医药公司 与酪氨酰-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
AU2011301804B2 (en) 2010-09-16 2015-07-16 Gen-Probe Incorporated Capture probes immobilizable via L-nucleotide tail
WO2012040124A1 (en) 2010-09-22 2012-03-29 Alios Biopharma, Inc. Azido nucleosides and nucleotide analogs
RU2624048C2 (ru) 2010-10-06 2017-06-30 Курна, Инк. Лечение заболеваний, связанных с геном сиалидазы 4 (neu4), путем ингибирования природного антисмыслового транскрипта гена neu4
US20140031250A1 (en) 2010-10-07 2014-01-30 David Tsai Ting Biomarkers of Cancer
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
JP6049623B2 (ja) 2010-10-22 2016-12-21 カッパーアールエヌエー,インコーポレイテッド α‐L‐イズロニダーゼ(IDUA)への天然アンチセンス転写物の阻害によるIDUA関連疾患の治療
ES2663009T3 (es) 2010-10-29 2018-04-10 Sirna Therapeutics, Inc. Inhibición de la expresión génica mediada por interferencia por ARN utilizando ácidos nucleicos de interferencia cortos (ANic)
EP3266881B1 (en) 2010-11-05 2019-12-25 Genalyte, Inc. Optical analyte detection systems and methods of use
US9150864B2 (en) 2010-11-08 2015-10-06 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
EP2638163B1 (en) 2010-11-12 2017-05-17 The General Hospital Corporation Polycomb-associated non-coding rnas
EP3521451A1 (en) 2010-11-17 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
WO2012068340A2 (en) 2010-11-18 2012-05-24 Opko Curna Llc Antagonat compositions and methods of use
CN103459599B (zh) 2010-11-23 2017-06-16 库尔纳公司 通过抑制nanog的天然反义转录物而治疗nanog相关疾病
DK2646550T3 (da) 2010-12-02 2015-05-26 Keygene Nv Målrettet ændring af DNA med oligonukleotider
EP2857512B1 (en) 2010-12-02 2016-06-29 Keygene N.V. Targeted alteration of DNA
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHOD FOR INCREASING AN ERYTHROPOIETIN (EPO) PREPARATION
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
US9045749B2 (en) 2011-01-14 2015-06-02 The General Hospital Corporation Methods targeting miR-128 for regulating cholesterol/lipid metabolism
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
RU2702501C2 (ru) 2011-03-29 2019-10-08 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии гена tmprss6
EA201792185A3 (ru) 2011-04-01 2018-10-31 Айонис Фармасьютикалз, Инк. Модуляция экспрессии трансдуктора сигнала и активатора транскрипции 3 (stat3)
KR101839177B1 (ko) 2011-04-13 2018-03-15 아이오니스 파마수티컬즈, 인코포레이티드 Ptpib 발현의 안티센스 조절
RS61447B1 (sr) 2011-04-21 2021-03-31 Glaxo Group Ltd Modulacija ekspresije virusa hepatitisa b (hbv)
RU2016138742A (ru) 2011-04-27 2018-12-12 Айсис Фармасьютикалс, Инк. МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА СIII (АроСIII)
WO2012151324A1 (en) 2011-05-02 2012-11-08 Isis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
RU2620980C2 (ru) 2011-06-09 2017-05-30 Курна, Инк. Лечение заболеваний, связанных с фратаксином (fxn), путем ингибирования природного антисмыслового транскрипта fxn
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
EP3320922A1 (en) 2011-06-10 2018-05-16 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
US9187749B2 (en) 2011-06-10 2015-11-17 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
JP6043347B2 (ja) 2011-06-16 2016-12-14 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 線維芽細胞増殖因子受容体4の発現のアンチセンス調節
KR20230107908A (ko) 2011-06-21 2023-07-18 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(ANGPTL3) iRNA 조성물 및 그사용 방법
EP2723390B1 (en) 2011-06-23 2017-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
WO2013003808A1 (en) 2011-06-29 2013-01-03 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
EP2732035A2 (en) 2011-07-15 2014-05-21 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
US9885082B2 (en) 2011-07-19 2018-02-06 University Of Idaho Embodiments of a probe and method for targeting nucleic acids
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
WO2013022990A1 (en) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2013033223A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
EP3453761A1 (en) 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2753317B1 (en) 2011-09-06 2020-02-26 CuRNA, Inc. TREATMENT OF DISEASES RELATED TO ALPHA SUBUNITS OF SODIUM CHANNELS, VOLTAGE-GATED (SCNxA) WITH SMALL MOLECULES
US9644241B2 (en) 2011-09-13 2017-05-09 Interpace Diagnostics, Llc Methods and compositions involving miR-135B for distinguishing pancreatic cancer from benign pancreatic disease
CN108410868A (zh) 2011-09-20 2018-08-17 Ionis制药公司 Gcgr表达的反义调节
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
AU2012322788B2 (en) 2011-10-11 2018-01-04 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
BR112014009790A2 (pt) 2011-10-25 2018-05-15 Isis Pharmaceuticals Inc composto para modulação antisense da expressão de gccr, seu uso e composição
WO2013067050A1 (en) 2011-10-31 2013-05-10 University Of Utah Research Foundation Genetic alterations in glioblastoma
EP2776564B1 (en) 2011-11-07 2019-10-02 Ionis Pharmaceuticals, Inc. Modulation of tmprss6 expression
US9243291B1 (en) 2011-12-01 2016-01-26 Isis Pharmaceuticals, Inc. Methods of predicting toxicity
TR201809173T4 (tr) 2011-12-08 2018-07-23 Sarepta Therapeutics Inc İnsan LMNA'sını hedef alan oligonükleotid analogları.
US20130156849A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
US9546368B2 (en) 2011-12-22 2017-01-17 Ionis Pharmaceuticals, Inc. Methods for modulating metastasis-associated-in-lung-adenocarcinoma-transcript-1 (MALAT-1) expression
PT2794627T (pt) 2011-12-22 2018-12-19 Janssen Biopharma Inc Nucleósidos substituídos, nucleótidos e análogos dos mesmos
EP2797948B1 (en) 2011-12-27 2019-02-20 The J. David Gladstone Institutes Compositions for use in regulating glucose metabolism
ES2842938T3 (es) 2012-01-11 2021-07-15 Ionis Pharmaceuticals Inc Composiciones y métodos para la modulación del empalme de IKBKAP
WO2013120003A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
EP2825885B1 (en) 2012-03-12 2021-05-12 The Board of Trustees of the University of Illinois Optical analyte detection systems with magnetic enhancement
CN104583405A (zh) 2012-03-15 2015-04-29 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
US9441007B2 (en) 2012-03-21 2016-09-13 Alios Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
USRE48171E1 (en) 2012-03-21 2020-08-25 Janssen Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9192651B2 (en) 2012-04-02 2015-11-24 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP3336189A1 (en) 2012-04-20 2018-06-20 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
EP2852606B1 (en) 2012-05-22 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
PL2855500T3 (pl) 2012-05-24 2020-11-30 Ionis Pharmaceuticals, Inc. Sposoby i kompozycje do modulowania ekspresji apolipoproteiny(a)
EA027929B1 (ru) 2012-05-25 2017-09-29 Янссен Сайенсиз Айрлэнд Юси Нуклеозиды на основе урацила и спирооксетана
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
EP3461895B1 (en) 2012-06-25 2020-07-01 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
EP2877579B1 (en) 2012-07-27 2019-12-18 Ionis Pharmaceuticals, Inc. Modulation of renin-angiotensin system (ras) related diseases by angiotensinogen
WO2014028739A1 (en) 2012-08-15 2014-02-20 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified capping protocols
WO2014045126A2 (en) 2012-09-18 2014-03-27 Uti Limited Partnership Treatment of pain by inhibition of usp5 de-ubiquitinase
EP4052709A1 (en) 2012-10-11 2022-09-07 Ionis Pharmaceuticals, Inc. Methods of treating kennedy's disease
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
WO2014059353A2 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
US20150275208A1 (en) 2012-10-12 2015-10-01 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
MX370924B (es) 2012-10-15 2020-01-09 Ionis Pharmaceuticals Inc Composiciones para modular la expresión de c9orf72.
US10577604B2 (en) 2012-10-15 2020-03-03 Ionis Pharmaceuticals, Inc. Methods for monitoring C9ORF72 expression
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
RU2689548C2 (ru) 2012-10-31 2019-05-28 Ионис Фармасьютикалз, Инк. Лечение рака
US20150299803A1 (en) 2012-11-05 2015-10-22 Pronai Therapeutics, Inc. Methods of Using Biomarkers for the Treatment of Cancer by Modulation of BCL2 Expression
AU2013337277B2 (en) 2012-11-05 2018-03-08 Foundation Medicine, Inc. Novel NTRK1 fusion molecules and uses thereof
DK2920304T3 (da) 2012-11-15 2019-05-13 Roche Innovation Ct Copenhagen As Oligonukleotidkonjugater
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
US9725723B2 (en) 2012-11-26 2017-08-08 Roche Innovation Center Copenhagen A/S Compositions and methods for modulation of FGFR3 expression
WO2014093537A1 (en) 2012-12-11 2014-06-19 Isis Pharmaceuticals, Inc. Competitive modulation of micrornas
LT2935303T (lt) 2012-12-21 2021-03-25 Janssen Biopharma, Inc. 4'-fluor-nukleozidai, 4'-fluor-nukleotidai ir jų analogai, skirti hcv gydymui
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
CA3150658A1 (en) 2013-01-18 2014-07-24 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
RU2649367C2 (ru) 2013-01-30 2018-04-02 Ф. Хоффманн-Ля Рош Аг Конъюгаты углевода и lna-олигонуклеотида
EP2951191B1 (en) 2013-01-31 2018-10-17 Ionis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
EP3778618A1 (en) 2013-02-04 2021-02-17 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CN104994884B (zh) 2013-02-14 2018-07-17 Ionis制药公司 对脂蛋白脂肪酶缺乏(lpld)群体中的载脂蛋白c-iii(apociii)表达的调节
WO2014130922A1 (en) 2013-02-25 2014-08-28 Trustees Of Boston University Compositions and methods for treating fungal infections
WO2014134144A1 (en) 2013-02-28 2014-09-04 The General Hospital Corporation Mirna profiling compositions and methods of use
WO2014134179A1 (en) 2013-02-28 2014-09-04 The Board Of Regents Of The University Of Texas System Methods for classifying a cancer as susceptible to tmepai-directed therapies and treating such cancers
WO2014159813A1 (en) 2013-03-13 2014-10-02 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
PT2970974T (pt) 2013-03-14 2017-11-29 Alnylam Pharmaceuticals Inc Composições de irna de componente do complemento c5 e métodos para a sua utilização
US9273349B2 (en) 2013-03-14 2016-03-01 Affymetrix, Inc. Detection of nucleic acids
WO2014153236A1 (en) 2013-03-14 2014-09-25 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9983206B2 (en) 2013-03-15 2018-05-29 The Board Of Trustees Of The University Of Illinois Methods and compositions for enhancing immunoassays
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
CN105378085B (zh) 2013-05-01 2019-02-15 Ionis制药公司 用于调节hbv和ttr表达的组合物和方法
IL285780B (en) 2013-05-22 2022-07-01 Alnylam Pharmaceuticals Inc Preparations of tmprss6 irna and methods of using them
SG11201509712YA (en) 2013-05-22 2016-01-28 Alnylam Pharmaceuticals Inc SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20160113911A1 (en) 2013-06-06 2016-04-28 The General Hospital Corporation Methods and compositions for the treatment of cancer
EP3011028B1 (en) 2013-06-21 2019-06-12 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
WO2014205449A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
RS59986B1 (sr) 2013-06-27 2020-03-31 Roche Innovation Ct Copenhagen As Antisens oligomeri i konjugati koji ciljno deluju na pcsk9
ES2787600T3 (es) 2013-07-02 2020-10-16 Ionis Pharmaceuticals Inc Moduladores del receptor de la hormona del crecimiento
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
EP3022176B8 (en) 2013-07-15 2019-12-25 The Regents of the University of California Azacyclic constrained analogs of fty720
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ma公司 用於調節τ蛋白表現之組合物
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
AU2014306271A1 (en) 2013-08-08 2016-03-24 The Scripps Research Institute A method for the site-specific enzymatic labelling of nucleic acids in vitro by incorporation of unnatural nucleotides
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
WO2015031679A2 (en) 2013-08-28 2015-03-05 Isis Pharmaceuticals, Inc. Modulation of prekallikrein (pkk) expression
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
WO2015034928A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
KR20210088009A (ko) 2013-09-05 2021-07-13 사렙타 쎄러퓨틱스, 인코퍼레이티드 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함
EP3043827B1 (en) 2013-09-13 2019-07-03 Ionis Pharmaceuticals, Inc. Modulators of complement factor b
US9943604B2 (en) 2013-09-20 2018-04-17 Ionis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
WO2015050990A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CA2926218A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
CA2925357C (en) 2013-10-04 2024-02-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
EP3055414A4 (en) 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US10053692B2 (en) 2013-10-21 2018-08-21 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
US9758546B2 (en) 2013-10-21 2017-09-12 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
WO2015075166A1 (en) 2013-11-22 2015-05-28 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for treatment of a bacterial infection
US20170268000A1 (en) 2013-12-02 2017-09-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
MX2016007409A (es) 2013-12-12 2016-12-14 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (ari) contra el componente del complemento y metodos para su uso.
AU2014364520B2 (en) 2013-12-20 2020-01-02 The General Hospital Corporation Methods and assays relating to circulating tumor cells
KR102298476B1 (ko) 2013-12-24 2021-09-03 아이오니스 파마수티컬즈, 인코포레이티드 안지오포이에틴-유사 3 발현의 조절
EP3099798B1 (en) 2014-01-29 2018-06-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Oligonucleotides and methods for inhibiting or reducing bacterial biofilms
JP6594902B2 (ja) 2014-02-11 2019-10-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法
EP3119789B1 (en) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
RU2702838C2 (ru) 2014-03-19 2019-10-11 Ионис Фармасьютикалз, Инк. Композиции для модуляции экспрессии атаксина 2
ES2818236T3 (es) 2014-04-01 2021-04-09 Biogen Ma Inc Composiciones para modular la expresión de SOD-1
US10513706B2 (en) 2014-04-09 2019-12-24 The Scripps Research Institute Import of unnatural or modified nucleoside triphosphates into cells via nucleic acid triphosphate transporters
WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10221416B2 (en) 2014-04-24 2019-03-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
EP3647318B1 (en) 2014-04-28 2021-06-30 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
EP3974534A1 (en) 2014-05-01 2022-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
PL3137605T3 (pl) 2014-05-01 2021-04-19 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji białka angiopoetynopodobnego 3
EA036496B1 (ru) 2014-05-01 2020-11-17 Ионис Фармасьютикалз, Инк. Конъюгированные олигонуклеотиды для модулирования экспрессии фактора комплемента b
JP6665111B2 (ja) 2014-05-01 2020-03-13 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. Pkk発現を調節するための組成物及び方法
EP3811977A1 (en) 2014-05-01 2021-04-28 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
WO2015175510A1 (en) 2014-05-12 2015-11-19 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
KR20220087576A (ko) 2014-05-22 2022-06-24 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐 (agt) irna 조성물 및 이의 사용 방법
CA2950589A1 (en) 2014-06-02 2015-12-10 Children's Medical Center Corporation Methods and compositions for immunomodulation
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2015200697A1 (en) 2014-06-25 2015-12-30 The General Hospital Corporation Targeting human satellite ii (hsatii)
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
JP6940404B2 (ja) 2014-08-19 2021-09-29 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 核酸検出のための方法および組成物
US10385343B2 (en) 2014-08-29 2019-08-20 Children's Medical Center Corporation Methods and compositions for the treatment of cancer
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
WO2016053638A1 (en) * 2014-09-30 2016-04-07 Ge Healthcare Bio-Sciences Corp. Method for nucleic acid analysis directly from an unpurified biological sample
SG11201702682PA (en) 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3206751A4 (en) 2014-10-14 2018-06-13 The J. David Gladstone Institutes Compositions and methods for reactivating latent immunodeficiency virus
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
WO2016077704A1 (en) 2014-11-14 2016-05-19 The Regents Of The University Of California Modulation of agpat5 expression
JP2017535552A (ja) 2014-11-17 2017-11-30 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. アポリポタンパク質C3(APOC3)iRNA組成物およびその使用方法
EP3221445B1 (en) 2014-11-20 2021-07-14 The Regents of The University of California Compositions and methods related to hematologic recovery
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
CA2970177C (en) 2014-12-08 2023-09-19 The Board Of Regents Of The University Of Texas System Lipocationic polymers and uses thereof
AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
AU2016219263B2 (en) 2015-02-13 2022-12-01 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
CA2977965C (en) 2015-02-26 2021-12-21 Ionis Pharmaceuticals, Inc. Allele specific modulators of p23h rhodopsin
ES2909308T3 (es) 2015-03-03 2022-05-06 Ionis Pharmaceuticals Inc Métodos para modular la expresión de MECP2
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3265098A4 (en) 2015-03-03 2019-02-13 Ionis Pharmaceuticals, Inc. COMPOSITIONS FOR MODULATING MECP2 EXPRESSION
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016161429A1 (en) 2015-04-03 2016-10-06 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating tmprss6 expression
WO2016164463A1 (en) 2015-04-07 2016-10-13 The General Hospital Corporation Methods for reactivating genes on the inactive x chromosome
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
NZ733882A (en) 2015-04-16 2020-04-24 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
EP3302497A4 (en) 2015-06-01 2019-01-16 Sarepta Therapeutics, Inc. EXCLUSION OF EXON INDUCED PAT TECHNOLOGY ANTISENSE IN COLLAGEN TYPE VII
EP4365291A3 (en) 2015-06-12 2024-08-14 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
WO2016210241A1 (en) 2015-06-26 2016-12-29 Beth Israel Deaconess Medical Center, Inc. Cancer therapy targeting tetraspanin 33 (tspan33) in myeloid derived suppressor cells
WO2017004261A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
CN109477106B (zh) 2015-07-10 2022-10-04 Ionis制药公司 二酰基甘油酰基转移酶2(dgat2)的调节剂
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
AU2016295168B2 (en) 2015-07-17 2021-08-19 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
US11273167B2 (en) 2015-08-03 2022-03-15 The Regents Of The University Of California Compositions and methods for modulating ABHD2 activity
CN108366966A (zh) 2015-08-24 2018-08-03 光环生物干扰疗法公司 用于调节基因表达的多核苷酸纳米颗粒及其用途
DK3341479T3 (da) 2015-08-24 2020-02-24 Roche Innovation Ct Copenhagen As LNA-G-Proces
US10889813B2 (en) 2015-09-02 2021-01-12 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
DK3349802T3 (en) 2015-09-14 2021-10-11 Univ Texas Lipocationic dendrimers and uses thereof
WO2017048843A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
EP3352753A4 (en) 2015-09-24 2019-03-13 The Regents of The University of California SYNTHETIC SPHINGOLIPIDE-SIMILAR MOLECULES, MEDICAMENTS, METHODS FOR THEIR SYNTHESIS AND TREATMENT PROCEDURES
CN108513588A (zh) 2015-09-24 2018-09-07 Ionis制药公司 Kras表达的调节剂
PT3353303T (pt) 2015-09-25 2023-10-10 Academisch Ziekenhuis Leiden Composições e métodos para modulação da expressão de ataxina 3
SG10202003148VA (en) 2015-10-08 2020-05-28 Ionis Pharmaceuticals Inc Compounds and methods for modulating angiotensinogen expression
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017068087A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Oligonucleotide detection method
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
PT4119569T (pt) 2015-11-06 2024-10-23 Ionis Pharmaceuticals Inc Compostos antissentido conjugados para utilização em terapia
PE20181180A1 (es) 2015-11-06 2018-07-20 Ionis Pharmaceuticals Inc MODULAR LA EXPRESION DE APOLIPOPROTEINA (a)
SI4220360T1 (sl) 2015-11-12 2024-10-30 F. Hoffmann - La Roche Ag Oligonukleotidi za indukcijo očetovske ekspresije UBE3A
CN108699556B (zh) 2015-11-16 2023-02-17 奥利克斯医药有限公司 使用靶向myd88或tlr3的rna复合物治疗老年性黄斑变性
CN109072235B (zh) 2015-11-23 2023-02-28 加利福尼亚大学董事会 通过核递送crispr/cas9追踪并操纵细胞rna
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. METHODS OF TREATING BREAST CANCER
IL259795B2 (en) 2015-12-07 2024-04-01 Genzyme Corp Methods and preparations for the treatment of diseases related to SERPINC1
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
WO2017120365A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
KR20180104075A (ko) 2016-02-02 2018-09-19 올릭스 주식회사 IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
EP3419665B1 (en) 2016-02-25 2024-10-30 The Brigham and Women's Hospital, Inc. Treatment methods for fibrosis targeting smoc2
US11136577B2 (en) 2016-03-09 2021-10-05 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
ES2857702T3 (es) 2016-03-14 2021-09-29 Hoffmann La Roche Oligonucleótidos para la reducción de la expresión de PD-L1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
CA3013799A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Methods of modulating keap1
WO2017180835A1 (en) 2016-04-13 2017-10-19 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
EP3442983A1 (en) 2016-04-14 2019-02-20 H. Hoffnabb-La Roche Ag TRITYL-MONO-Ga1NAc COMPOUNDS AND THEIR USE
AU2017254106B2 (en) 2016-04-18 2024-07-11 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
US10822608B2 (en) 2016-04-29 2020-11-03 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human LMNA
EP3449002A4 (en) 2016-04-29 2019-09-25 Nanyang Technological University ANTISENSE OLIGONUCLEOTIDES CONTAINING A G-QUADRUPLEX
BR112018072362A2 (pt) 2016-05-06 2019-02-19 Ionis Pharmaceuticals, Inc. oligonucleotídeos conjugados a uma unidade de ligando do receptor glp-1 e seus usos
CN114656378A (zh) 2016-05-16 2022-06-24 得克萨斯州大学系统董事会 阳离子磺酰胺氨基脂质和两亲性两性离子氨基脂质
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
DK3464306T3 (da) 2016-05-24 2024-05-21 Sarepta Therapeutics Inc Fremgangsmåder til fremstilling af phosphorodiamidat-morpholino-oligomerer
KR102506298B1 (ko) 2016-05-24 2023-03-06 사렙타 쎄러퓨틱스 인코퍼레이티드 올리고머의 제조 방법
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
WO2017205506A1 (en) 2016-05-24 2017-11-30 Emory University Particles with rna cleaving nucleobase polymers and uses for managing inflammatory disorders
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
MA45496A (fr) 2016-06-17 2019-04-24 Hoffmann La Roche Molécules d'acide nucléique pour la réduction de l'arnm de padd5 ou pad7 pour le traitement d'une infection par l'hépatite b
WO2017219017A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
JP7074345B2 (ja) 2016-06-22 2022-05-24 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 一本鎖rna編集オリゴヌクレオチド
DK3475295T3 (da) 2016-06-24 2022-10-24 Scripps Research Inst Hidtil ukendt nukleosidtriphosphat-transportør og anvendelser deraf
EP3478697A1 (en) 2016-06-30 2019-05-08 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
AU2017289204A1 (en) 2016-07-01 2019-01-17 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating HTRA1 expression
PL3484524T3 (pl) 2016-07-15 2023-03-20 Ionis Pharmaceuticals, Inc. Związki i sposoby modulacji smn2
WO2018017814A1 (en) 2016-07-20 2018-01-25 President And Fellows Of Harvard College Peptidoglycan glycosyltransferase inhibitors of sed proteins for treating bacterial infections
DK3507366T3 (da) 2016-09-01 2020-11-02 Proqr Therapeutics Ii Bv Kemisk modificeret, enkeltstrengede rna-modificerende oligonukleotider
WO2018053185A1 (en) 2016-09-14 2018-03-22 Alios Biopharma, Inc. Modified oligonucleotides and methods of use
US11202818B2 (en) 2016-09-14 2021-12-21 President And Fellows Of Harvard College Methods and compositions for modulating erythropoiesis
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
EP3532638A4 (en) 2016-10-31 2020-07-29 University of Massachusetts TARGETING MICROARN-101-3 P IN CARCINOTHERAPY
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
JP7033591B2 (ja) 2016-11-11 2022-03-10 ロシュ イノベーション センター コペンハーゲン エーエス 治療用オリゴヌクレオチドの捕捉および検出
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
EP3548620A4 (en) 2016-12-02 2020-07-22 Cold Spring Harbor Laboratory MODULATION OF THE EXPRESSION OF LNC05
WO2018112032A1 (en) 2016-12-13 2018-06-21 President And Fellows Of Harvard College Methods and compositions for targeting tumor-infiltrating tregs using inhibitors of ccr8 and tnfrsf8
EP3555292A1 (en) 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
EP4122497B1 (en) 2016-12-19 2024-04-10 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
HUE059843T2 (hu) 2016-12-19 2023-01-28 Sarepta Therapeutics Inc Exonátugró oligomerkonjugátumok izomdisztrófiára
MD3554552T2 (ro) 2016-12-19 2022-12-31 Sarepta Therapeutics Inc Conjugați oligomeri de omitere a exonilor pentru distrofie musculară
US20190345495A1 (en) 2017-01-13 2019-11-14 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb2 expression
WO2018130582A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rel expression
EP3568477A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rela expression
WO2018130583A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb1 expression
EP3568481A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating relb expression
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals Morpholino modified oligomeric compounds
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
CN110913898B (zh) 2017-04-18 2024-04-05 阿尔尼拉姆医药品有限公司 具有乙肝病毒(hbv)感染的受试者的治疗方法
AU2018256435A1 (en) 2017-04-20 2019-11-07 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
US11453891B2 (en) 2017-05-10 2022-09-27 The Regents Of The University Of California Directed editing of cellular RNA via nuclear delivery of CRISPR/CAS9
US20190055564A1 (en) 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
AU2018300069A1 (en) 2017-07-11 2020-02-27 Synthorx, Inc. Incorporation of unnatural nucleotides and methods thereof
EP3661955A4 (en) 2017-08-03 2021-05-26 Synthorx, Inc. CYTOKINE CONJUGATES FOR THE TREATMENT OF AUTOIMMUNE DISEASES
WO2019030313A2 (en) 2017-08-11 2019-02-14 Roche Innovation Center Copenhagen A/S OLIGONUCLEOTIDES FOR MODULATION OF UBE3C EXPRESSION
AU2018318231A1 (en) 2017-08-18 2020-02-13 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
WO2019038228A1 (en) 2017-08-22 2019-02-28 Roche Innovation Center Copenhagen A/S OLIGONUCLEOTIDES FOR MODULATION OF TOM1 EXPRESSION
WO2019040923A1 (en) 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
CN118345073A (zh) 2017-09-14 2024-07-16 詹森生物制药有限公司 GalNAc衍生物
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP2020536057A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための併用療法
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
JP2021502059A (ja) 2017-10-13 2021-01-28 ロシュ イノベーション センター コペンハーゲン エーエス 部分的に立体定義されたオリゴヌクレオチドのサブライブラリーを使用することによる、アンチセンスオリゴヌクレオチドの、改良された立体定義されたホスホロチオエートオリゴヌクレオチド変異体を同定するための方法
BR112020007417A2 (pt) 2017-10-16 2021-01-26 F. Hoffmann-La Roche Ag molécula de ácido nucleico para redução de papd5 e papd7 de mrna para o tratamento de infecção hepatite b
EP3697904A1 (en) 2017-10-17 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment for cystic fibrosis
EP3697910A4 (en) 2017-10-18 2021-07-14 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERIC COMPOUNDS
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
WO2019115416A2 (en) 2017-12-11 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating fndc3b expression
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
JP2021506239A (ja) 2017-12-14 2021-02-22 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 複合アンチセンス化合物及びその使用
MX2020006012A (es) 2017-12-18 2020-09-14 Alnylam Pharmaceuticals Inc Composiciones de arni de la caja 1 del grupo de alta movilidad (hmgb1) y métodos de uso de las mismas.
WO2019121838A1 (en) 2017-12-21 2019-06-27 F. Hoffmann-La Roche Ag Companion diagnostic for htra1 rna antagonists
US11459564B2 (en) 2017-12-21 2022-10-04 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
MX2020005754A (es) 2017-12-22 2020-08-20 Roche Innovation Ct Copenhagen As Oligonucleotidos gapmeros que comprenden un enlace internucleosido fosforoditioato.
EP4092118A1 (en) 2017-12-22 2022-11-23 Roche Innovation Center Copenhagen A/S Novel thiophosphoramidites
CN111757936A (zh) 2017-12-22 2020-10-09 罗氏创新中心哥本哈根有限公司 包含二硫代磷酸酯核苷间连接的寡核苷酸
US20210095274A1 (en) 2018-01-10 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating pias4 expression
CN111836624A (zh) 2018-01-12 2020-10-27 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
US20210095275A1 (en) 2018-01-12 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating gsk3b expression
MX2020006973A (es) 2018-01-12 2020-09-09 Roche Innovation Ct Copenhagen As Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos.
BR112020014011A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso dirigidos à alfa-sinucleína e usos dos mesmos
US20200392510A1 (en) 2018-01-15 2020-12-17 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US20210095276A1 (en) 2018-01-17 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating erc1 expression
JP2021511029A (ja) 2018-01-18 2021-05-06 ロシュ イノベーション センター コペンハーゲン エーエス Srebp1を標的とするアンチセンスオリゴヌクレオチド
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
CR20200346A (es) 2018-02-09 2020-10-19 Genentech Inc Oligonucleótidos para modular la expresión de tmem106b
JP7317029B2 (ja) 2018-02-12 2023-07-28 アイオーニス ファーマシューティカルズ, インコーポレーテッド 修飾化合物及びその使用
CN112020559A (zh) 2018-02-21 2020-12-01 百时美施贵宝公司 Camk2d反义寡核苷酸及其用途
AR114445A1 (es) 2018-02-26 2020-09-09 Synthorx Inc Conjugados de il-15, y sus usos
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
EP3765476A2 (en) 2018-03-13 2021-01-20 Janssen Pharmaceutica NV Modified oligonucleotides for use in treatment of tauopathies
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
AU2019247645A1 (en) 2018-04-05 2020-10-15 Centre Leon Berard Use of FUBP1 inhibitors for treating hepatitis B virus infection
CA3094020A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US20220002796A1 (en) 2018-05-07 2022-01-06 Roche Innovation Center Copenhagen A/S Quality control of lna oligonucleotide therapeutics using massively parallel sequencing
JP7353301B2 (ja) 2018-05-07 2023-09-29 アルニラム ファーマスーティカルズ インコーポレイテッド 肝臓外送達
US20210371860A1 (en) 2018-05-08 2021-12-02 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
SG11202010012PA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing fxi expression
CN112189053B (zh) 2018-05-09 2024-05-14 Ionis制药公司 用于减少atxn3表达的化合物和方法
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
US20210220387A1 (en) 2018-05-18 2021-07-22 Hoffmann-La Roche, Inc. Pharmaceutical compositions for treatment of microrna related diseases
WO2019224172A1 (en) 2018-05-25 2019-11-28 Roche Innovation Center Copenhagen A/S Novel process for making allofuranose from glucofuranose
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2019233921A1 (en) 2018-06-05 2019-12-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating atxn2 expression
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
US11833168B2 (en) 2018-06-14 2023-12-05 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
CN112334776B (zh) 2018-06-21 2024-07-09 豪夫迈·罗氏有限公司 杂交全lna寡核苷酸
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
CR20210058A (es) 2018-07-03 2021-03-22 Hoffmann La Roche Oligonucleótidos para modular la expresión de tau
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020011653A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020011743A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mafb
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
CR20210015A (es) 2018-07-13 2021-03-22 Hoffmann La Roche Oligonucleótidos para modular la expresión de rtel 1
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
MX2020013270A (es) 2018-07-31 2021-02-18 Roche Innovation Ct Copenhagen As Oligonucleotidos que comprenden enlace internucleosidico de fosforotritioato.
EP4122943B1 (en) 2018-07-31 2024-05-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
MX2021001056A (es) 2018-08-13 2021-04-12 Alnylam Pharmaceuticals Inc Composiciones de agente de acido ribonucleico bicatenario (arnbc) de virus de la hepatitis b (vhb) y metodos de uso de las mismas.
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
WO2020041348A1 (en) 2018-08-20 2020-02-27 Rogcon, Inc. Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies
WO2020038971A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting vcan
WO2020038973A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting sptlc1
WO2020038968A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
CN113728102A (zh) 2018-08-28 2021-11-30 罗氏创新中心哥本哈根有限公司 使用剪接调节化合物进行新抗原工程化
WO2020047229A1 (en) 2018-08-29 2020-03-05 University Of Massachusetts Inhibition of protein kinases to treat friedreich ataxia
EP3620519A1 (en) 2018-09-04 2020-03-11 F. Hoffmann-La Roche AG Use of isolated milk extracellular vesicles for delivering oligonucleotides orally
US11273137B2 (en) 2018-09-04 2022-03-15 Board Of Trustees Of Michigan State University Methods and compositions to prevent and treat disorders associated with mutations in the ODC1 gene
US20210332359A1 (en) 2018-09-07 2021-10-28 The General Hospital Corporation Compositions and methods for immune checkpoint inhibition
EP3620520A1 (en) 2018-09-10 2020-03-11 Universidad del Pais Vasco Novel target to treat a metabolic disease in an individual
US20210332367A1 (en) 2018-09-18 2021-10-28 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
TW202423454A (zh) 2018-09-19 2024-06-16 美商Ionis製藥公司 Pnpla3表現之調節劑
WO2020069055A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
EP3873920A1 (en) 2018-11-01 2021-09-08 F. Hoffmann-La Roche AG Antisense oligonucleotides targeting tia1
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
EA202191342A1 (ru) 2018-11-15 2021-08-10 Айонис Фармасьютикалз, Инк. Модуляторы экспрессии irf5
KR20210093970A (ko) 2018-11-21 2021-07-28 아이오니스 파마수티컬즈, 인코포레이티드 프리온 발현을 감소시키기 위한 화합물 및 방법
WO2020104492A1 (en) 2018-11-22 2020-05-28 Roche Innovation Center Copenhagen A/S Pyridinium salts as activators in the synthesis of stereodefined oligonucleotides
WO2020109343A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Combination therapy for treatment of macular degeneration
WO2020109344A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Occular administration device for antisense oligonucleotides
KR20210104759A (ko) 2018-12-13 2021-08-25 사렙타 쎄러퓨틱스 인코퍼레이티드 근 이영양증을 위한 엑손 스키핑 올리고머 접합체
KR20210104555A (ko) 2018-12-17 2021-08-25 일루미나 케임브리지 리미티드 서열분석을 위한 프라이머 올리고뉴클레오타이드
JP7513616B2 (ja) 2018-12-20 2024-07-09 ヴィア・バイオテクノロジー・インコーポレイテッド 併用hbv療法
WO2020132521A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
EP3898975A2 (en) 2018-12-21 2021-10-27 Boehringer Ingelheim International GmbH Antisense oligonucleotides targeting card9
JP2022517270A (ja) 2019-01-16 2022-03-07 ジェンザイム・コーポレーション Serpinc1 iRNA組成物およびその使用方法
WO2020152303A1 (en) 2019-01-25 2020-07-30 F. Hoffmann-La Roche Ag Lipid vesicle for oral drug delivery
AU2020216444A1 (en) 2019-01-31 2021-07-29 Ionis Pharmaceuticals, Inc. Modulators of YAP1 expression
CA3127689A1 (en) 2019-02-06 2020-08-13 Synthorx, Inc. Il-2 conjugates and methods of use thereof
WO2020169696A1 (en) 2019-02-20 2020-08-27 Roche Innovation Center Copenhagen A/S Novel phosphoramidites
EP3927826A1 (en) 2019-02-20 2021-12-29 Roche Innovation Center Copenhagen A/S Phosphonoacetate gapmer oligonucleotides
EP3931348B1 (en) 2019-02-26 2023-08-09 Roche Innovation Center Copenhagen A/S Oligonucleotide formulation method
JP7492526B2 (ja) 2019-02-27 2024-05-29 アイオーニス ファーマシューティカルズ, インコーポレーテッド Malat1発現のモジュレーター
EP3934695A1 (en) 2019-03-05 2022-01-12 F. Hoffmann-La Roche AG Intracellular targeting of molecules
AU2020237225A1 (en) 2019-03-12 2021-10-14 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2020191177A1 (en) 2019-03-21 2020-09-24 Sudhir Agrawal Antisense oligonucleotides for allele specificity
MX2021011242A (es) 2019-03-21 2022-01-19 Codiak Biosciences Inc Conjugados de vesícula extracelular y usos de estos.
CA3135180A1 (en) 2019-03-29 2020-10-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating ube3a-ats
US20220213484A1 (en) 2019-04-03 2022-07-07 Bristol-Myers Squibb Company Angptl2 antisense oligonucleotides and uses thereof
JP7499267B2 (ja) 2019-04-04 2024-06-13 エフ. ホフマン-ラ ロシュ アーゲー Atxn2発現を調節するためのオリゴヌクレオチド
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
CN113661168A (zh) 2019-04-16 2021-11-16 罗氏创新中心哥本哈根有限公司 用于制备核苷酸p(v)单体的新方法
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
JP2022530537A (ja) 2019-04-30 2022-06-29 ロシュ イノベーション センター コペンハーゲン エーエス レニウムキレート化mag3オリゴヌクレオチドを調製するための新規の方法
KR20220004675A (ko) 2019-05-03 2022-01-11 다이서나 파마수이티컬, 인크. 단축 센스 가닥을 갖는 이중 가닥 핵산 억제제 분자
AU2020276243A1 (en) 2019-05-13 2021-11-25 Vir Biotechnology, Inc. Compositions and methods for treating hepatitis B virus (HBV) infection
WO2020236600A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
TW202111123A (zh) 2019-05-28 2021-03-16 美商Ionis製藥公司 用於減少fus 表現之化合物及方法
CN113950529A (zh) 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
EP3980554A1 (en) 2019-06-07 2022-04-13 F. Hoffmann-La Roche AG Hybridizing all-lna oligonucleotides
WO2020252257A1 (en) 2019-06-12 2020-12-17 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
JP2022538784A (ja) 2019-06-14 2022-09-06 ザ スクリプス リサーチ インスティテュート 半合成生物における複製、転写および翻訳のための試薬ならびに方法
EP3997225A1 (en) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
EP3956450A4 (en) 2019-07-26 2022-11-16 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATION OF GFAP
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
EP4013457A1 (en) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extracellular vesicle linked to molecules and uses thereof
JP2022544288A (ja) 2019-08-14 2022-10-17 コディアック バイオサイエンシーズ, インコーポレイテッド CEBP/βを標的とする細胞外小胞-ASO構築物
KR20220070433A (ko) 2019-08-14 2022-05-31 코디악 바이오사이언시즈, 인크. Stat6을 표적으로 하는 세포외 소포-aso 작제물
CA3147701A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicles with antisense oligonucleotides targeting kras
WO2021030768A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicles with stat3-antisense oligonucleotides
EP4013876A1 (en) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extracellular vesicle-nlrp3 antagonist
CN114555128A (zh) 2019-08-15 2022-05-27 新索思股份有限公司 采用il-2缀合物的免疫肿瘤学组合疗法
EP4013767A4 (en) 2019-08-15 2023-10-25 Ionis Pharmaceuticals, Inc. COMPOUND-MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
US20210054040A1 (en) 2019-08-23 2021-02-25 Synthorx, Inc. Novel il-15 conjugates and uses thereof
EP4025694A1 (en) 2019-09-03 2022-07-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
TW202124385A (zh) 2019-09-10 2021-07-01 美商欣爍克斯公司 治療自體免疫疾病之il-2接合物及使用方法
WO2021062058A1 (en) 2019-09-25 2021-04-01 Codiak Biosciences, Inc. Sting agonist comprising exosomes for treating neuroimmunological disorders
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
JP2022552249A (ja) 2019-10-14 2022-12-15 アストラゼネカ・アクチエボラーグ Pnpla3発現のモジュレーター
WO2021074657A1 (en) 2019-10-17 2021-04-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment for cystic fibrosis
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
MX2022004726A (es) 2019-10-22 2022-05-13 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de componente c3 de complemento y metodos de uso de las mismas.
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
MX2022004388A (es) 2019-11-01 2022-05-06 Alnylam Pharmaceuticals Inc Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas.
KR20220097445A (ko) 2019-11-04 2022-07-07 신톡스, 인크. 인터류킨 10 접합체 및 이의 용도
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
JP2023500681A (ja) 2019-11-06 2023-01-10 アルニラム ファーマスーティカルズ インコーポレイテッド 肝臓外送達
WO2021096763A1 (en) 2019-11-13 2021-05-20 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
EP4061943A1 (en) 2019-11-19 2022-09-28 Institut National de la Santé et de la Recherche Médicale (INSERM) Antisense oligonucleotides and their use for the treatment of cancer
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
KR20220115995A (ko) 2019-12-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 9 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
EP4077344A1 (en) 2019-12-18 2022-10-26 F. Hoffmann-La Roche AG Methods of sequencing by synthesis using a consecutive labeling scheme
EP4077667A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of sept9 inhibitors for treating hepatitis b virus infection
WO2021122869A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of scamp3 inhibitors for treating hepatitis b virus infection
WO2021122921A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
CN114829601A (zh) 2019-12-19 2022-07-29 豪夫迈·罗氏有限公司 Sbds抑制剂用于治疗乙型肝炎病毒感染的用途
EP4077671A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of saraf inhibitors for treating hepatitis b virus infection
CN114829603A (zh) 2019-12-20 2022-07-29 豪夫迈·罗氏有限公司 用于抑制scn9a表达的增强寡核苷酸
CN114828852A (zh) 2019-12-24 2022-07-29 豪夫迈·罗氏有限公司 用于治疗hbv的靶向hbv的抗病毒药剂和/或免疫调节剂的药物组合
JP2023509870A (ja) 2019-12-24 2023-03-10 エフ. ホフマン-ラ ロシュ アーゲー Hbvの処置のためのhbvを標的とする治療用オリゴヌクレオチドとtlr7アゴニストとの医薬組合せ
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
WO2021158810A1 (en) 2020-02-05 2021-08-12 Bristol-Myers Squibb Company Oligonucleotides for splice modulation of camk2d
TW202140786A (zh) 2020-02-10 2021-11-01 美商艾爾妮蘭製藥公司 用於靜默vegf-a表現之組合物及方法
CA3171648A1 (en) 2020-02-18 2021-08-26 Gilead Sciences, Inc. Antiviral compounds
KR20220143106A (ko) 2020-02-18 2022-10-24 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 C3 (APOC3) iRNA 조성물 및 이의 사용 방법
TW202245800A (zh) 2020-02-18 2022-12-01 美商基利科學股份有限公司 抗病毒化合物
TWI794742B (zh) 2020-02-18 2023-03-01 美商基利科學股份有限公司 抗病毒化合物
CN115176010A (zh) 2020-02-28 2022-10-11 豪夫迈·罗氏有限公司 用于调节cd73外显子7剪接的寡核苷酸
CA3173034A1 (en) 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
JP2023515671A (ja) 2020-03-04 2023-04-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 免疫療法に対する腫瘍細胞の感作のための方法及び組成物
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
JP2023516095A (ja) 2020-03-06 2023-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
JP2023516748A (ja) 2020-03-06 2023-04-20 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(ttr)の発現を阻害するための組成物および方法
WO2021184020A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Methods of treating neuroinflammation
WO2021184021A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting pmp22
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021198883A1 (en) 2020-03-31 2021-10-07 Janssen Biopharma, Inc. Synthesis of oligonucleotides and related compounds
KR20230008729A (ko) 2020-04-06 2023-01-16 알닐람 파마슈티칼스 인코포레이티드 Myoc 발현을 사일런싱하기 위한 조성물 및 방법
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
TW202204617A (zh) 2020-04-07 2022-02-01 美商艾爾妮蘭製藥公司 用於靜默scn9a表現之組合物及方法
WO2021222065A1 (en) 2020-04-27 2021-11-04 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
EP4142738A1 (en) 2020-04-30 2023-03-08 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
JP2023524065A (ja) 2020-05-01 2023-06-08 アイオーニス ファーマシューティカルズ, インコーポレーテッド Atxn1を調節するための化合物及び方法
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
EP4149486A1 (en) 2020-05-11 2023-03-22 Genentech, Inc. Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
WO2021231204A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same
EP4150085A1 (en) 2020-05-11 2023-03-22 Genentech, Inc. Complement component c1r inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
JP2023526267A (ja) 2020-05-13 2023-06-21 エフ. ホフマン-ラ ロシュ アーゲー プログラニュリン標的オリゴヌクレオチドアゴニスト
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
CA3162416C (en) 2020-05-15 2023-07-04 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
US20230193269A1 (en) 2020-05-22 2023-06-22 Boehringer Ingelheim International Gmbh Oligonucleotides for splice modulation of card9
US20230285357A1 (en) 2020-05-27 2023-09-14 The Regents Of The University Of California Compositions and methods for transdifferentiating cells
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
JP2023529457A (ja) 2020-06-09 2023-07-10 ロシュ イノベーション センター コペンハーゲン エーエス 治療用ポリヌクレオチドに使用するためのグアノシン類似体
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
EP4168546A1 (en) 2020-06-18 2023-04-26 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
EP4171747A1 (en) 2020-06-24 2023-05-03 VIR Biotechnology, Inc. Engineered hepatitis b virus neutralizing antibodies and uses thereof
CA3183834A1 (en) 2020-06-25 2021-12-30 Giovanni Abbadessa Immuno oncology combination therapy with il-2 conjugates and anti-egfr antibodies
AR122731A1 (es) 2020-06-26 2022-10-05 Hoffmann La Roche Oligonucleótidos mejorados para modular la expresión de fubp1
JP2023532518A (ja) 2020-06-29 2023-07-28 アイオーニス ファーマシューティカルズ, インコーポレーテッド Plp1を調節するための化合物及び方法
EP4179091A1 (en) 2020-07-10 2023-05-17 Institut National De La Sante Et De La Recherche Medicale - Inserm Methods and compositions for treating epilepsy
WO2022011214A1 (en) 2020-07-10 2022-01-13 Alnylam Pharmaceuticals, Inc. Circular sirnas
WO2022018155A1 (en) 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
EP4185696A1 (en) 2020-07-23 2023-05-31 F. Hoffmann-La Roche AG Oligonucleotides targeting rna binding protein sites
JP2023538630A (ja) 2020-08-21 2023-09-08 エフ. ホフマン-ラ ロシュ アーゲー B型肝炎ウイルス感染症を処置するためのa1cf阻害剤の使用
WO2022056454A2 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Methods and compositions for treating hpv-positive cancers
WO2022060986A2 (en) 2020-09-16 2022-03-24 President And Fellows Of Harvard College Methods of treating an individual that has failed an anti-pd-1/anti-pd-l1 therapy
TW202227102A (zh) 2020-09-22 2022-07-16 瑞典商阿斯特捷利康公司 治療脂肪肝病之方法
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
JP2023544413A (ja) 2020-10-05 2023-10-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド Gタンパク質共役受容体75(GPR75)iRNA組成物およびその使用方法
US20230364127A1 (en) 2020-10-06 2023-11-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
IL301612A (en) 2020-10-09 2023-05-01 Synthorx Inc Immuno-oncological treatments with IL-2 couples
AU2021356693A1 (en) 2020-10-09 2023-06-15 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and pembrolizumab
EP4232581A1 (en) 2020-10-21 2023-08-30 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
TW202237841A (zh) 2020-11-13 2022-10-01 美商艾拉倫製藥股份有限公司 凝血因子V(F5)iRNA組成物及其使用方法
MX2023005736A (es) 2020-11-18 2023-05-25 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la expresion de angiotensinogeno.
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
AR124227A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
JP2023553069A (ja) 2020-12-08 2023-12-20 エフ. ホフマン-ラ ロシュ アーゲー ホスホロジチオエートオリゴヌクレオチドの新規合成
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
CA3205040A1 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
TW202229553A (zh) 2020-12-18 2022-08-01 瑞士商赫孚孟拉羅股份公司 靶向顆粒蛋白前體之反義寡核苷酸
JP2024501662A (ja) 2020-12-22 2024-01-15 エフ. ホフマン-ラ ロシュ アーゲー Xbp1を標的とするオリゴヌクレオチド
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
WO2022147214A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
TW202246500A (zh) 2021-02-02 2022-12-01 瑞士商赫孚孟拉羅股份公司 用於抑制 rtel1 表現之增強型寡核苷酸
TW202302148A (zh) 2021-02-12 2023-01-16 美商欣爍克斯公司 使用il-2接合物和抗pd-1抗體或其抗原結合片段的肺癌組合療法
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
TW202245843A (zh) 2021-02-12 2022-12-01 美商欣爍克斯公司 Il-2接合物及西米普利單抗(cemiplimab)之皮膚癌組合療法
EP4294456A1 (en) 2021-02-17 2023-12-27 Lonza Sales AG Extracellular vesicle linked to a biologically active molecule via an optimized linker and an anchoring moiety
JP2024512236A (ja) 2021-02-17 2024-03-19 ロンザ セールス アーゲー 細胞外ベシクル-nlrp3アンタゴニスト
JP2024509783A (ja) 2021-02-25 2024-03-05 アルナイラム ファーマシューティカルズ, インコーポレイテッド プリオンタンパク質(prnp)irna組成物およびその使用方法
KR20230150844A (ko) 2021-02-26 2023-10-31 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나아제(KHK) iRNA 조성물 및 이의 사용 방법
IL305442A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
JP2024512635A (ja) 2021-03-29 2024-03-19 アルナイラム ファーマシューティカルズ, インコーポレイテッド ハンチンチン(HTT)iRNA剤組成物およびその使用方法
WO2022241408A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating tissue distribution of intracellular therapeutics
EP4314016A1 (en) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
JP2024513841A (ja) 2021-04-01 2024-03-27 ロンザ セールス アーゲー 細胞外小胞組成物
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
JP2024512771A (ja) 2021-04-16 2024-03-19 ギリアード サイエンシーズ, インコーポレイテッド アミドを使用してカルバヌクレオシドを調製する方法
AU2022264478A1 (en) 2021-04-26 2023-10-12 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
CA3217463A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for modulating mrna splicing
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
WO2022246023A1 (en) 2021-05-20 2022-11-24 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
TW202313679A (zh) 2021-06-03 2023-04-01 美商欣爍克斯公司 包含il-2接合物及pd-1拮抗劑之頭頸癌組合療法
KR20240017911A (ko) 2021-06-04 2024-02-08 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 9 개방 해독 프레임 72(C9orf72) iRNA 제제 조성물 및 이의 사용 방법
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
WO2022258555A1 (en) 2021-06-08 2022-12-15 F. Hoffmann-La Roche Ag Oligonucleotide progranulin agonists
US11753644B2 (en) 2021-06-18 2023-09-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing IFNAR1 expression
KR20240037226A (ko) 2021-06-22 2024-03-21 아큐라스템 인코포레이티드 Pikfyve 안티센스 올리고뉴클레오티드
IL309001A (en) 2021-06-23 2024-02-01 Entrada Therapeutics Inc Antisense compounds and methods for targeting CUG repeats
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
EP4363580A1 (en) 2021-06-30 2024-05-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
EP4367237A2 (en) 2021-07-09 2024-05-15 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
JP2024528659A (ja) 2021-07-19 2024-07-30 アルナイラム ファーマシューティカルズ, インコーポレイテッド 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物
JP2024526890A (ja) 2021-07-21 2024-07-19 アルナイラム ファーマシューティカルズ, インコーポレイテッド 代謝障害関連標的遺伝子irna組成物およびその使用方法
AU2022316149A1 (en) 2021-07-21 2024-02-08 AcuraStem Incorporated Unc13a antisense oligonucleotides
WO2023003995A1 (en) 2021-07-23 2023-01-26 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
JP2024529437A (ja) 2021-07-29 2024-08-06 アルナイラム ファーマシューティカルズ, インコーポレイテッド 3-ヒドロキシ-3-メチルグリタル-COAレダクターゼ(HMGCR)iRNA組成物およびその使用方法
JP2024530018A (ja) 2021-08-03 2024-08-14 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(TTR)iRNA組成物およびその使用方法
EP4381071A1 (en) 2021-08-04 2024-06-12 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
JP2024534766A (ja) 2021-08-13 2024-09-26 アルナイラム ファーマシューティカルズ, インコーポレイテッド 第XII因子(F12)iRNA組成物およびその使用方法
WO2023021046A1 (en) 2021-08-16 2023-02-23 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
US12116380B2 (en) 2021-08-18 2024-10-15 Gilead Sciences, Inc. Phospholipid compounds and methods of making and using the same
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
WO2023034817A1 (en) 2021-09-01 2023-03-09 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 44 in duchenne muscular dystrophy
JP2024535850A (ja) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ, インコーポレイテッド 補体成分(C3)をサイレンシングするためのiRNA組成物および方法
KR20240083183A (ko) 2021-09-20 2024-06-11 알닐람 파마슈티칼스 인코포레이티드 인히빈 서브유닛 베타 e (inhbe) 조절제 조성물 및 이의 이용 방법
EP4408999A1 (en) 2021-09-29 2024-08-07 F. Hoffmann-La Roche AG Rna editing
IL311568A (en) 2021-09-30 2024-05-01 Sarepta Therapeutics Inc Antisense oligonucleotides having one or more basic units
IL311518A (en) 2021-10-01 2024-05-01 Adarx Pharmaceuticals Inc Preparations that modulate perkalkerine and methods of using them
EP4416284A1 (en) 2021-10-15 2024-08-21 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
JP2024539223A (ja) 2021-10-22 2024-10-28 サレプタ セラピューティクス, インコーポレイテッド 末梢ミエリンタンパク質22関連疾患の処置のためのモルフォリノオリゴマー
EP4419683A1 (en) 2021-10-22 2024-08-28 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2023076451A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023078883A1 (en) 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
EP4444881A2 (en) 2021-11-10 2024-10-16 University of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
KR20240101580A (ko) 2021-11-11 2024-07-02 에프. 호프만-라 로슈 아게 Hbv 치료를 위한 약학 조합물
WO2023091704A1 (en) 2021-11-18 2023-05-25 Circularis Biotechnologies, Inc. Compositions and methods for production of circular nucleic acid molecules
US20230159930A1 (en) 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof
EP4437106A2 (en) 2021-11-24 2024-10-02 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
WO2023104693A1 (en) 2021-12-07 2023-06-15 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting actl6b
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
EP4448106A1 (en) 2021-12-17 2024-10-23 Hoffmann-La Roche Inc. Combination of oligonucleotides for modulating rtel1 and fubp1
WO2023111336A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Oligonucleotide gba agonists
CN118434860A (zh) 2021-12-20 2024-08-02 豪夫迈·罗氏有限公司 苏糖核酸反义寡核苷酸及其方法
EP4452327A1 (en) 2021-12-20 2024-10-30 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
EP4453211A1 (en) 2021-12-22 2024-10-30 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
CN118613268A (zh) 2022-01-20 2024-09-06 基因泰克公司 用于调节tmem106b表达的反义寡核苷酸
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2023152369A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Nucleic acid mir-9 inhibitor for the treatment of cystic fibrosis
US20240167040A1 (en) 2022-02-21 2024-05-23 Hoffmann-La Roche Inc. Antisense oligonucleotide
WO2023212625A1 (en) 2022-04-28 2023-11-02 AcuraStem Incorporated Syf2 antisense oligonucleotides
US20240002857A1 (en) 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
WO2023217890A1 (en) 2022-05-10 2023-11-16 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting cfp-elk1 intergene region
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
TW202409276A (zh) 2022-05-18 2024-03-01 瑞士商赫孚孟拉羅股份公司 改良之靶向rna結合蛋白位點的寡核苷酸
WO2023240277A2 (en) 2022-06-10 2023-12-14 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
WO2023242324A1 (en) 2022-06-17 2023-12-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for targeting progranulin
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024031101A1 (en) 2022-08-05 2024-02-08 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
WO2024040041A1 (en) 2022-08-15 2024-02-22 Dicerna Pharmaceuticals, Inc. Regulation of activity of rnai molecules
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
TW202424186A (zh) 2022-08-25 2024-06-16 美商生命編輯治療學公司 Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾
WO2024050261A1 (en) 2022-08-29 2024-03-07 University Of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
EP4332221A1 (en) 2022-08-29 2024-03-06 Roche Innovation Center Copenhagen A/S Threose nucleic acid antisense oligonucleotides and methods thereof
TW202421206A (zh) 2022-09-06 2024-06-01 瑞士商赫孚孟拉羅股份公司 用於投予至眼睛之雙股rna分子
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
WO2024081954A2 (en) 2022-10-14 2024-04-18 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
WO2024098061A2 (en) 2022-11-04 2024-05-10 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
WO2024098002A1 (en) 2022-11-04 2024-05-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
US20240173426A1 (en) 2022-11-14 2024-05-30 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024119145A1 (en) 2022-12-01 2024-06-06 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2024126654A1 (en) 2022-12-14 2024-06-20 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting actl6b
WO2024137590A2 (en) 2022-12-19 2024-06-27 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
WO2024136899A1 (en) 2022-12-21 2024-06-27 Synthorx, Inc. Cancer therapy with il-2 conjugates and chimeric antigen receptor therapies
WO2024146935A1 (en) 2023-01-06 2024-07-11 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
WO2024155986A2 (en) 2023-01-20 2024-07-25 AcuraStem Incorporated Unc13a antisense oligonucleotides
WO2024160756A1 (en) 2023-01-30 2024-08-08 Vib Vzw Suppressors of tauopathies
WO2024163651A2 (en) 2023-01-31 2024-08-08 AcuraStem Incorporated Syf2 antisense oligonucleotides
WO2024167945A1 (en) 2023-02-06 2024-08-15 AcuraStem Incorporated Pikfyve antisense oligonucleotides
WO2024168010A2 (en) 2023-02-09 2024-08-15 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof
WO2024175588A1 (en) 2023-02-21 2024-08-29 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
WO2024175586A2 (en) 2023-02-21 2024-08-29 Vib Vzw Inhibitors of synaptogyrin-3 expression
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
WO2024196937A1 (en) 2023-03-20 2024-09-26 Synthorx, Inc. Cancer therapy with il-2 peg conjugates
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024220930A2 (en) 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024227040A1 (en) 2023-04-27 2024-10-31 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of chronic kidney disease
WO2024227047A2 (en) 2023-04-28 2024-10-31 Beam Therapeutics Inc. Modified guide rna

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4837312A (en) * 1985-01-25 1989-06-06 California Institute Of Technology Chelator-functionalized nucleosides and nucleotides and methods for making same
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
EP1331011A3 (en) 1991-10-24 2003-12-17 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5559101A (en) * 1994-10-24 1996-09-24 Genencor International, Inc. L-ribofuranosyl nucleosides
JP2002503212A (ja) 1996-10-16 2002-01-29 アイ・シー・エヌ・フアーマシユーテイカルズ・インコーポレイテツド 単環式ヌクレオシド、その類似体および使用
AU4966997A (en) 1996-11-18 1998-06-10 Takeshi Imanishi Novel nucleotide analogues
JP3756313B2 (ja) * 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
AU9063398A (en) * 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6436640B1 (en) * 1999-03-18 2002-08-20 Exiqon A/S Use of LNA in mass spectrometry
US6525191B1 (en) * 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides

Also Published As

Publication number Publication date
CA2372085A1 (en) 2000-11-09
DE60033927T2 (de) 2007-11-29
WO2000066604A2 (en) 2000-11-09
CA2372085C (en) 2009-10-27
PT1178999E (pt) 2007-06-26
US20030087230A1 (en) 2003-05-08
KR20020013515A (ko) 2002-02-20
EP1178999A2 (en) 2002-02-13
KR100782896B1 (ko) 2007-12-06
JP2002543214A (ja) 2002-12-17
NZ514348A (en) 2004-05-28
DE60033927D1 (de) 2007-04-26
IL145497A0 (en) 2002-06-30
CN1349541A (zh) 2002-05-15
WO2000066604A3 (en) 2001-01-11
CN102180924A (zh) 2011-09-14
ATE356824T1 (de) 2007-04-15
US7053207B2 (en) 2006-05-30
ES2283298T3 (es) 2007-11-01
HK1048322A1 (zh) 2003-03-28
AU776362B2 (en) 2004-09-09
EP1178999B1 (en) 2007-03-14
AU4391800A (en) 2000-11-17

Similar Documents

Publication Publication Date Title
DK1178999T3 (da) L-RIBO-LNA-analoger
DE60041072D1 (de) Em nachweis
DE69920477D1 (de) 2'-modifizierte oligonucleotide mit rns wirkung, die konformell eingeschränkt sind
DE69806003D1 (de) Dna polymerasen, die verbesserte fähigkeiten zum einbau markierter nukleotide besitzen
ATE373671T1 (de) Aminooxy-modifizierte oligonukleotide
WO2001038584A3 (en) Non-aggregating, non-quenching oligomers comprising nucleotide analogues; methods of synthesis and use thereof
CY1120186T1 (el) Τροποποιημενα νουκλεοτιδια για αλληλουχιση πολυνουκλεοτιδιωn
HU9501963D0 (en) 7-deazapurine modified oligonucleotides
WO2001047944A3 (en) Nucleic acids containing single nucleotide polymorphisms and methods of use thereof
DE60325016D1 (de) Oligonukleotide mit alternierenden segmenten und deren verwendungen
ES2118593T3 (es) Analogos de nucleotidos de pteridina como sondas fluorescentes de adn.
ATE244259T1 (de) Verwendungen von fluoreszierenden n-nukleosiden und dessen analogen
AU2496900A (en) Enhanced sequencing by hybridization using pools of probes
ATE239090T1 (de) Methode zum generieren von einzelsträngigen dna molekülen
AU2002365811A1 (en) Methods for the selection and cloning of nucleic acid molecules free of unwanted nucleotide sequence alterations
ATE242261T1 (de) Fluoreszend-konjugaten von nukleosiden und von nukleotiden, ihrer verfahren zur herstellung und ihrer verwendungen
AU2002305073A1 (en) Synthesis of selenium-derivatized nucleosides, nucleotides, phosphoramidites, triphosphates and nucleic acids
HUP0300219A2 (hu) L-nukleozidokat, L-nukleotidokat és analógjaikat tartalmazó készítmények és alkalmazásuk
AU2003247592A8 (en) Selective cloning of homoduplex nucleic acids
WO2001048245A3 (en) Nucleic acids containing single nucleotide polymorphisms and methods of use thereof
WO2001040521A3 (en) Nucleic acids containing single nucleotide polymorphisms and methods of use thereof
WO2004083155A3 (en) Nucleotide lipid ester derivatives
WO2001023618A3 (en) Compositions and methods for reducing oligonucleotide hybridization and priming specificity
WO2003044168A3 (en) Facilitation of rna interference
WO2000029622A3 (en) Nucleic acids containing single nucleotide polymorphisms and methods of use thereof