LUC00046I1 - - Google Patents

Info

Publication number
LUC00046I1
LUC00046I1 LU00046C LUC00046C LUC00046I1 LU C00046 I1 LUC00046 I1 LU C00046I1 LU 00046 C LU00046 C LU 00046C LU C00046 C LUC00046 C LU C00046C LU C00046 I1 LUC00046 I1 LU C00046I1
Authority
LU
Luxembourg
Application number
LU00046C
Original Assignee
Cold Spring Harbor Laboratory
Biogen Ma Inc
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
Application filed by Cold Spring Harbor Laboratory, Biogen Ma Inc filed Critical Cold Spring Harbor Laboratory
Publication of LUC00046I1 publication Critical patent/LUC00046I1/fr
Publication of LUC00046I2 publication Critical patent/LUC00046I2/fr

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
LU00046C 2005-06-23 2017-11-07 LUC00046I2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23
EP06773838A EP1910395B1 (fr) 2005-06-23 2006-06-23 Compositions et procédés de modulation de l épissage de SMN2

Publications (2)

Publication Number Publication Date
LUC00046I1 true LUC00046I1 (fr) 2017-11-10
LUC00046I2 LUC00046I2 (fr) 2018-01-10

Family

ID=37595872

Family Applications (1)

Application Number Title Priority Date Filing Date
LU00046C LUC00046I2 (fr) 2005-06-23 2017-11-07

Country Status (13)

Country Link
US (6) US8361977B2 (fr)
EP (5) EP1910395B1 (fr)
CY (2) CY2017040I2 (fr)
DK (3) DK2548560T3 (fr)
ES (3) ES2702531T3 (fr)
HK (2) HK1181307A1 (fr)
HU (2) HUE027486T2 (fr)
LT (2) LT3308788T (fr)
LU (1) LUC00046I2 (fr)
PL (3) PL2548560T3 (fr)
PT (3) PT1910395E (fr)
SI (2) SI3308788T1 (fr)
WO (1) WO2007002390A2 (fr)

Families Citing this family (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
ES2702531T3 (es) 2005-06-23 2019-03-01 Biogen Ma Inc Composiciones y procedimientos para modulación de splicing de SMN2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2219680A2 (fr) 2007-11-13 2010-08-25 Isis Pharmaceuticals, Inc. Composés et méthodes destinées à la modulation de l'expression de protéines
WO2009151546A2 (fr) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Procédés de traitement d'une atrophie musculaire spinale
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
EP2370451B1 (fr) 2008-12-02 2016-11-16 Wave Life Sciences Japan, Inc. Procédé pour la synthèse d'acides nucléiques modifiés au niveau de l'atome de phosphore
IT1398361B1 (it) * 2009-01-21 2013-02-22 Fond Santa Lucia Uso di mutanti dominanti negativi di sam68 per il trattamento della sma
PT2417257E (pt) * 2009-04-10 2016-06-03 Universität Bern Oligonucleótidos triciclo-dna anti-sentido, composições, e métodos para o tratamento de doença
US20120149757A1 (en) * 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
MX2011014004A (es) * 2009-06-17 2012-06-19 Isis Pharmaceuticals Inc Composiciones y metodos para la modulacion de division de smn2 en un sujeto.
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
EP2576574A2 (fr) 2010-05-28 2013-04-10 Sarepta Therapeutics, Inc. Analogues oligonucléotidiques ayant des liaisons modifiées entre sous-unités et/ou des groupes terminaux modifiés
US9518259B2 (en) * 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012031243A2 (fr) 2010-09-03 2012-03-08 Avi Biopharma, Inc. Molécules d'arnds comportant des analogues oligonucléotidiques ayant des liaisons entre sous-unités et/ou des groupes terminaux modifiés
WO2012039448A1 (fr) 2010-09-24 2012-03-29 株式会社キラルジェン Groupe auxiliaire asymétrique
IT1405793B1 (it) * 2010-10-15 2014-01-24 Univ Ferrara Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica
EP2638163B1 (fr) 2010-11-12 2017-05-17 The General Hospital Corporation Arn non codants associés à polycomb
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
IL273838B (en) 2011-05-05 2022-09-01 Sarepta Therapeutics Inc Peptide and oligonucleotide conjugates, preparations containing them and their uses
WO2012178122A2 (fr) * 2011-06-23 2012-12-27 Cold Spring Harbor Laboratory Modèle phénocopique de maladie
AU2012284265B2 (en) 2011-07-19 2017-08-17 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CA2855241A1 (fr) 2011-11-11 2013-05-16 Santaris Pharma A/S Composes pour la modulation de l'epissage de smn2
MX354940B (es) 2011-11-18 2018-03-27 Sarepta Therapeutics Inc Oligonucleotidos funcionalmente modificados y subunidades de los mismos.
US9944926B2 (en) 2011-11-30 2018-04-17 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
EP3858847A1 (fr) 2011-11-30 2021-08-04 Sarepta Therapeutics, Inc. Oligonucléotides pour le traitement de maladies à répétition nucléotidique étendue
WO2013086207A1 (fr) * 2011-12-06 2013-06-13 The Ohio State University Agents de contraste non ioniques de faible osmolarité pour l'administration d'oligonucléotides antisens et le traitement de maladie
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CA2873797A1 (fr) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions et methodes pour moduler l'expression de utrn
CN104540947A (zh) 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节smn基因家族表达的组合物和方法
CA2873779A1 (fr) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions et methodes pour moduler l'expression de mecp2
CA2879023C (fr) 2012-07-13 2017-03-28 Wave Life Sciences Japan Groupe auxiliaire asymetrique
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
EP2943225A4 (fr) * 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc Compositions et procédés pour la modulation de l'épissage de smn2 chez un sujet
EP2946013A1 (fr) * 2013-01-16 2015-11-25 Iowa State University Research Foundation, Inc. Cible intronique profonde pour la correction d'épissure sur le gène de l'amyotrophie spinale (sma)
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
KR20210088009A (ko) 2013-09-05 2021-07-13 사렙타 쎄러퓨틱스, 인코퍼레이티드 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함
JP2016534035A (ja) 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
EP3095459A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
EP4137572A1 (fr) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Conception chirale
WO2015120450A1 (fr) * 2014-02-10 2015-08-13 Ohio State Innovation Foundation Oligonucléotides antisens dans le traitement de l'amyotrophie spinale
WO2015161170A2 (fr) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions et procédés de modulation de l'épissage du smn2 chez un patient
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
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
SG11201702682PA (en) 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
CA2966044A1 (fr) 2014-10-30 2016-05-06 The General Hospital Corporation Procedes de modulation de la repression genique dependant d'atrx
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US10851371B2 (en) 2015-04-10 2020-12-01 Ionis Pharmaceuticals, Inc. Modulation of SMN expression
EP3302497A4 (fr) 2015-06-01 2019-01-16 Sarepta Therapeutics, Inc. Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
AU2016317667A1 (en) * 2015-08-28 2018-03-22 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
US9669109B1 (en) 2015-11-24 2017-06-06 Universita Di Ferrara Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy
EP3933041B1 (fr) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Oligomères antisens destinés au traitement du retard mental autosomique dominante
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017136435A1 (fr) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Composés permettant de moduler l'expression de fc-epsilon-ri-bêta et utilisations correspondantes
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
MX2018015654A (es) 2016-06-14 2019-06-10 Biogen Ma Inc Cromatografía de interacción hidrofóbica para la purificación de oligonucleótidos.
WO2017218884A1 (fr) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinaisons pour la modulation de l'expression de smn
WO2017223258A1 (fr) 2016-06-24 2017-12-28 Biogen Ma Inc. Synthèse d'oligonucléotides thiolés sans étape de coiffage
EP3481432A4 (fr) 2016-07-05 2020-05-06 The U.S.A. as represented by the Secretary, Department of Health and Human Services Diagnostic de maladies associées au gène col6 et procédés de traitement associés
US20190321387A1 (en) * 2016-07-15 2019-10-24 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
EP4035659A1 (fr) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes destinés à l'administration d'agents thérapeutiques
JP7455579B2 (ja) 2017-02-28 2024-03-26 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
AU2018256435A1 (en) 2017-04-20 2019-11-07 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
WO2019032102A1 (fr) 2017-08-09 2019-02-14 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Adn en duplex, linéaire, à extrémités fermées de virus adéno-associé et utilisations correspondantes
WO2019040923A1 (fr) 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. Oligomères antisens pour le traitement d'états pathologiques et autres maladies
EP3694530A4 (fr) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
EP3697910A4 (fr) 2017-10-18 2021-07-14 Sarepta Therapeutics, Inc. Composés oligomères antisens
EP3707264A1 (fr) 2017-11-08 2020-09-16 Avexis Inc. Moyens et procédé de préparation de vecteurs viraux et leurs utilisations
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
WO2019147960A1 (fr) 2018-01-25 2019-08-01 Biogen Ma Inc. Méthodes de traitement de l'amyotrophie musculaire
US12060558B2 (en) * 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2019236949A1 (fr) 2018-06-08 2019-12-12 Avexis Inc. Dosage basé sur des cellules permettant de mesurer la puissance d'un produit médicamenteux
JP2022509372A (ja) 2018-10-29 2022-01-20 バイオジェン・エムエイ・インコーポレイテッド 血液脳関門輸送の促進のためのヒト化され安定化されたfc5バリアント
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
MX2021006359A (es) 2018-11-30 2021-08-11 Novartis Ag Vectores virales de vaa y sus usos.
CA3162618A1 (fr) 2019-03-20 2020-09-24 Peter Jungsoo PARK Therapie d'augmentation de la progranuline a base d'oligonucleotides anti-sens dans les maladies neurodegeneratives
JP2022531876A (ja) 2019-05-08 2022-07-12 バイオジェン・エムエイ・インコーポレイテッド オリゴヌクレオチドの収束液相合成
CR20220071A (es) * 2019-07-19 2022-04-22 Biogen Ma Inc Composiciones farmacéuticas y métodos para tratar o prevenir la atrofia muscular espinal
CA3173034A1 (fr) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Composes et procedes de modulation de smn2
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
WO2022241408A1 (fr) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions et procédés de modulation de la distribution tissulaire d'agents thérapeutiques intracellulaires
EP4422613A1 (fr) 2021-10-25 2024-09-04 Novartis AG Méthodes pour améliorer l'administration de virus adéno-associé (vaa)
FR3128873A1 (fr) 2021-11-10 2023-05-12 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale
WO2023240236A1 (fr) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
FR2645345A1 (fr) 1989-03-31 1990-10-05 Thomson Csf Procede de modulation dirigee de la composition ou du dopage de semi-conducteurs, notamment pour la realisation de composants electroniques monolithiques de type planar, utilisation et produits correspondants
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
CN1123038A (zh) 1993-05-11 1996-05-22 北卡罗来纳大学查珀尔希尔分校 阻止异常剪接的反义寡核苷酸及其使用方法
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
DK0804219T3 (da) 1994-02-28 2002-11-11 Agritech Technologies Ltd Inhibinpræparater og fremgangsmåder til anvendelse deraf
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
DE19906484A1 (de) * 1999-02-17 2000-09-07 Wegerer Kristina Elektrische Maschine
ES2283298T3 (es) 1999-05-04 2007-11-01 Santaris Pharma A/S Analogos de l-ribo-lna.
US6649411B2 (en) 1999-07-30 2003-11-18 The United States Of America As Represented By The Department Of Health And Human Services Methods of inhibiting cancer cells with ADNF III antisense oligonucleotides
US6770633B1 (en) 1999-10-26 2004-08-03 Immusol, Inc. Ribozyme therapy for the treatment of proliferative skin and eye diseases
US6962906B2 (en) 2000-03-14 2005-11-08 Active Motif Oligonucleotide analogues, methods of synthesis and methods of use
ATE544473T1 (de) 2000-11-09 2012-02-15 Cold Spring Harbor Lab Chimäre moleküle zur modulation der genexpression
US6376508B1 (en) * 2000-12-13 2002-04-23 Academia Sinica Treatments for spinal muscular atrophy
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
EP2213738B1 (fr) 2002-11-14 2012-10-10 Dharmacon, Inc. Molécules siRNA anti Bcl-2
WO2004113867A2 (fr) 2003-06-16 2004-12-29 University Of Massachusetts Analyse d'exon
GB0425625D0 (en) 2004-11-22 2004-12-22 Royal College Of Surgeons Ie Treatment of disease
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2702531T3 (es) 2005-06-23 2019-03-01 Biogen Ma Inc Composiciones y procedimientos para modulación de splicing de SMN2
US20120021515A1 (en) 2009-02-06 2012-01-26 Swayze Eric E Oligomeric compounds and methods
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
MX2011014004A (es) 2009-06-17 2012-06-19 Isis Pharmaceuticals Inc Composiciones y metodos para la modulacion de division de smn2 en un sujeto.

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EP1910395A4 (fr) 2010-09-22
DK3308788T3 (da) 2019-01-02
EP3470072A1 (fr) 2019-04-17
US20170088835A1 (en) 2017-03-30
EP3308788A1 (fr) 2018-04-18
US8946183B2 (en) 2015-02-03
LTPA2017037I1 (lt) 2017-11-27
PT1910395E (pt) 2012-12-03
PT2548560E (pt) 2015-09-22
SI3308788T1 (sl) 2019-01-31
US20180291376A1 (en) 2018-10-11
EP1910395B1 (fr) 2012-10-17
ES2397113T3 (es) 2013-03-04
EP3308788B1 (fr) 2018-10-24
EP1910395A2 (fr) 2008-04-16
US8361977B2 (en) 2013-01-29
ES2545223T3 (es) 2015-09-09
SI2548560T1 (sl) 2015-12-31
DK2548560T3 (en) 2015-07-13
US20200181613A1 (en) 2020-06-11
EP2548560A1 (fr) 2013-01-23
LT3308788T (lt) 2018-12-10
CY1121134T1 (el) 2019-12-11
DK1910395T3 (da) 2013-02-04
HK1181307A1 (en) 2013-11-08
WO2007002390A3 (fr) 2007-11-15
ES2702531T3 (es) 2019-03-01
EP2644700A1 (fr) 2013-10-02
US20100216238A1 (en) 2010-08-26
PL3308788T3 (pl) 2019-05-31
PL1910395T3 (pl) 2013-03-29
EP2548560B1 (fr) 2015-06-03
LTC2548560I2 (lt) 2018-11-12
HUS1700042I1 (hu) 2017-12-28
HK1247103B (zh) 2019-09-06
LUC00046I2 (fr) 2018-01-10
US20150353929A1 (en) 2015-12-10
WO2007002390A2 (fr) 2007-01-04
CY2017040I1 (el) 2018-04-04
US20130109091A1 (en) 2013-05-02
PL2548560T3 (pl) 2015-11-30
PT3308788T (pt) 2019-01-30
CY2017040I2 (el) 2018-04-04
HUE027486T2 (en) 2016-09-28

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