CO2021014747A2 - Métodos para el tratamiento de βeta-talasemia - Google Patents

Métodos para el tratamiento de βeta-talasemia

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Publication number
CO2021014747A2
CO2021014747A2 CONC2021/0014747A CO2021014747A CO2021014747A2 CO 2021014747 A2 CO2021014747 A2 CO 2021014747A2 CO 2021014747 A CO2021014747 A CO 2021014747A CO 2021014747 A2 CO2021014747 A2 CO 2021014747A2
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CO
Colombia
Prior art keywords
thalassemia
methods
beta
treatment
compositions
Prior art date
Application number
CONC2021/0014747A
Other languages
English (en)
Inventor
Iv Weston P Miller
John Tomaro
Sagar A Vaidya
Mark Walters
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Sangamo Therapeutics Inc
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Publication date
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of CO2021014747A2 publication Critical patent/CO2021014747A2/es

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Abstract

En la presente memoria se describen métodos y composiciones para tratar una beta-talasemia
CONC2021/0014747A 2019-04-02 2021-11-02 Métodos para el tratamiento de βeta-talasemia CO2021014747A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962828182P 2019-04-02 2019-04-02
US201962930846P 2019-11-05 2019-11-05
US201962944626P 2019-12-06 2019-12-06
PCT/US2020/025919 WO2020205838A1 (en) 2019-04-02 2020-03-31 Methods for the treatment of beta-thalassemia

Publications (1)

Publication Number Publication Date
CO2021014747A2 true CO2021014747A2 (es) 2021-11-19

Family

ID=70465393

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0014747A CO2021014747A2 (es) 2019-04-02 2021-11-02 Métodos para el tratamiento de βeta-talasemia

Country Status (15)

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US (2) US20200316116A1 (es)
EP (1) EP3946384A1 (es)
JP (1) JP2022519949A (es)
KR (1) KR20210146986A (es)
CN (2) CN113939586A (es)
AU (1) AU2020253362A1 (es)
BR (1) BR112021019448A2 (es)
CA (1) CA3132167A1 (es)
CO (1) CO2021014747A2 (es)
IL (1) IL286857A (es)
MA (1) MA55531A (es)
MX (1) MX2021012152A (es)
SG (1) SG11202110776TA (es)
TW (1) TW202102665A (es)
WO (1) WO2020205838A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023014839A1 (en) * 2021-08-03 2023-02-09 Sangamo Therapeutics, Inc. Methods for the treatment of sickle cell disease

Family Cites Families (100)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL47062A (en) 1975-04-10 1979-07-25 Yeda Res & Dev Process for diminishing antigenicity of tissues to be usedas transplants by treatment with glutaraldehyde
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4665077A (en) 1979-03-19 1987-05-12 The Upjohn Company Method for treating rejection of organ or skin grafts with 6-aryl pyrimidine compounds
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
IL85746A (en) 1988-03-15 1994-05-30 Yeda Res & Dev Preparations comprising t-lymphocyte cells treated with 8-methoxypsoralen or cell membranes separated therefrom for preventing or treating autoimmune diseases
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
WO1990008187A1 (en) 1989-01-19 1990-07-26 Dana Farber Cancer Institute Soluble two domain cd2 protein
ES2062519T5 (es) 1989-03-21 2003-07-16 Immune Response Corp Inc Vacunacion y metodos contra enfermedades originadas a partir de respuestas patogenicas mediante poblaciones especificas de linfocitos t.
DE69031919T3 (de) 1989-07-19 2005-01-27 Connetics Corp., Palo Alto T-zell-rezeptor-peptide als heilmittel für autoimmune und bösartige krankheiten
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5430057A (en) * 1993-09-30 1995-07-04 Board Of Regents, The University Of Texas System Parenteral busulfan for treatment of malignant disease
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
EP0770129B1 (en) 1994-01-18 2005-11-23 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
ES2316148T3 (es) 1994-03-23 2009-04-01 Ohio University Acidos nucleicos compactados y su suministro a celulas.
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
EP1060261B1 (en) 1998-03-02 2010-05-05 Massachusetts Institute of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US6379903B1 (en) 1999-10-08 2002-04-30 Sigma-Aldrich Co. Purification of recombinant proteins fused to multiple epitopes
WO2001040798A2 (en) 1999-12-06 2001-06-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
CA2398590C (en) 2000-02-08 2012-08-28 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US7030846B2 (en) 2001-07-10 2006-04-18 Samsung Electronics Co., Ltd. Color correction liquid crystal display and method of driving same
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Cnnについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
ATE531796T1 (de) 2002-03-21 2011-11-15 Sangamo Biosciences Inc Verfahren und zusammensetzungen zur verwendung von zinkfinger-endonukleasen zur verbesserung der homologen rekombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005100392A2 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
JP4903689B2 (ja) 2004-04-08 2012-03-28 サンガモ バイオサイエンシズ インコーポレイテッド 神経障害および神経変性症状を治療するための方法と組成物
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
US20090068164A1 (en) 2005-05-05 2009-03-12 The Ariz Bd Of Regents On Behald Of The Univ Of Az Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
EP1913149A4 (en) 2005-07-26 2009-08-05 Sangamo Biosciences Inc TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES
EP2650365B1 (en) 2005-10-18 2016-09-14 Precision Biosciences Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity
EP2213731B1 (en) 2006-05-25 2013-12-04 Sangamo BioSciences, Inc. Variant foki cleavage half-domains
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
DE102008025277A1 (de) 2008-05-27 2009-12-03 Merck Patent Gmbh Glaszusammensetzung
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
AU2011213242B2 (en) 2010-02-08 2015-01-29 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
LT2566972T (lt) 2010-05-03 2020-03-10 Sangamo Therapeutics, Inc. Kompozicijos, skirtos cinko pirštu modulių susiejimui
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
CA3157027A1 (en) 2010-07-21 2012-01-26 Sangamo Therapeutics, Inc. Methods and compositions for modification of a t-cell receptor gene
US9458205B2 (en) 2011-11-16 2016-10-04 Sangamo Biosciences, Inc. Modified DNA-binding proteins and uses thereof
CA3171494A1 (en) 2012-08-29 2014-03-06 Sangamo Biosciences, Inc. Cells genetically modified within the bcl11a gene by a nuclease and various aspects related thereto
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
LT3068881T (lt) 2013-11-13 2019-08-12 Children`S Medical Center Corporation Geno raiškos reguliavimas, tarpininkaujant nukleazei
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
PT3102673T (pt) 2014-02-03 2020-05-21 Sangamo Biosciences Inc Métodos e composições para tratamento de beta-talassemia
JP6722176B2 (ja) 2014-09-16 2020-07-15 サンガモ セラピューティクス, インコーポレイテッド 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物
MX2017014446A (es) 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
MX2018012729A (es) * 2016-04-18 2019-07-04 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de hemoglobinopatias.
ES2900317T3 (es) 2016-08-24 2022-03-16 Sangamo Therapeutics Inc Regulación de la expresión génica al usar nucleasas genomanipuladas
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors

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US20230381225A1 (en) 2023-11-30
CA3132167A1 (en) 2020-10-08
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