AU2017345430B2 - Methods and compositions for the treatment of Fabry disease - Google Patents

Methods and compositions for the treatment of Fabry disease Download PDF

Info

Publication number
AU2017345430B2
AU2017345430B2 AU2017345430A AU2017345430A AU2017345430B2 AU 2017345430 B2 AU2017345430 B2 AU 2017345430B2 AU 2017345430 A AU2017345430 A AU 2017345430A AU 2017345430 A AU2017345430 A AU 2017345430A AU 2017345430 B2 AU2017345430 B2 AU 2017345430B2
Authority
AU
Australia
Prior art keywords
variant
gla
donor
glako
cdna
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2017345430A
Other languages
English (en)
Other versions
AU2017345430A1 (en
Inventor
Marshall W. HUSTON
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics 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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of AU2017345430A1 publication Critical patent/AU2017345430A1/en
Application granted granted Critical
Publication of AU2017345430B2 publication Critical patent/AU2017345430B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2017345430A 2016-10-20 2017-10-19 Methods and compositions for the treatment of Fabry disease Active AU2017345430B2 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201662410543P 2016-10-20 2016-10-20
US62/410,543 2016-10-20
US201762444093P 2017-01-09 2017-01-09
US62/444,093 2017-01-09
US201762458324P 2017-02-13 2017-02-13
US62/458,324 2017-02-13
US201762502058P 2017-05-05 2017-05-05
US62/502,058 2017-05-05
US201762516373P 2017-06-07 2017-06-07
US62/516,373 2017-06-07
US201762552792P 2017-08-31 2017-08-31
US62/552,792 2017-08-31
PCT/US2017/057328 WO2018075736A1 (en) 2016-10-20 2017-10-19 Methods and compositions for the treatment of fabry disease

Publications (2)

Publication Number Publication Date
AU2017345430A1 AU2017345430A1 (en) 2019-05-02
AU2017345430B2 true AU2017345430B2 (en) 2024-09-05

Family

ID=62018856

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017345430A Active AU2017345430B2 (en) 2016-10-20 2017-10-19 Methods and compositions for the treatment of Fabry disease

Country Status (12)

Country Link
US (2) US11219695B2 (cg-RX-API-DMAC7.html)
EP (2) EP3528852B1 (cg-RX-API-DMAC7.html)
JP (2) JP7042263B2 (cg-RX-API-DMAC7.html)
KR (1) KR102712926B1 (cg-RX-API-DMAC7.html)
CN (1) CN110022904B (cg-RX-API-DMAC7.html)
AU (1) AU2017345430B2 (cg-RX-API-DMAC7.html)
BR (1) BR112019007210A2 (cg-RX-API-DMAC7.html)
CA (1) CA3039673A1 (cg-RX-API-DMAC7.html)
ES (1) ES3047063T3 (cg-RX-API-DMAC7.html)
IL (1) IL266047B2 (cg-RX-API-DMAC7.html)
MX (1) MX2019004487A (cg-RX-API-DMAC7.html)
WO (1) WO2018075736A1 (cg-RX-API-DMAC7.html)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3528852B1 (en) 2016-10-20 2025-09-03 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
US20220184185A1 (en) * 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
MX2021004278A (es) * 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para la expresion de transgenes a partir de un locus de albumina.
MX2021008131A (es) * 2019-01-04 2021-10-13 Sangamo Therapeutics Inc Metodos y composiciones para el tratamiento de la enfermedad de fabry.
JP7600124B2 (ja) * 2019-02-04 2024-12-16 フリーライン セラピューティクス リミテッド ポリヌクレオチド
MX2021011754A (es) * 2019-03-27 2021-10-22 Sigilon Therapeutics Inc Composiciones, dispositivos y metodos para el tratamiento de la enfermedad de fabry.
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
CN113058041B (zh) * 2020-08-27 2022-04-05 华东师范大学 一种用于治疗庞贝氏病的产品
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
KR102438536B1 (ko) * 2020-04-28 2022-09-05 케이비바이오메드 주식회사 경구용 핵산 분자 전달 복합체
WO2022015813A1 (en) * 2020-07-15 2022-01-20 The Jackson Laboratory Humanized mouse models for sars-cov-2 infection
WO2022155665A1 (en) * 2021-01-14 2022-07-21 Spark Therapeutics, Inc. Compositions and methods for treating fabry disease
KR20240099332A (ko) * 2021-11-03 2024-06-28 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 바이러스 벡터 작제물의 사용 방법

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140112896A1 (en) * 2012-07-11 2014-04-24 Sangqmo BioSciences, Inc. Methods and compositions for the provision of proteins deficient in lysosomal storage diseases
US20160060656A1 (en) * 2012-07-11 2016-03-03 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases

Family Cites Families (139)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
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
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
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
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
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
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (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
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US6544761B2 (en) * 1994-12-13 2003-04-08 Human Genome Sciences, Inc. Human tissue inhibitor of metalloproteinase-4
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
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
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
WO2000009153A1 (en) 1997-10-29 2000-02-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
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
US6479626B1 (en) 1998-03-02 2002-11-12 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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 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
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
EP1236045B1 (en) 1999-12-06 2005-11-09 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
US20030073652A1 (en) * 2000-05-17 2003-04-17 Harvey Pollard Ex-vivo and in vivo factor XII gene therapy for hemophilia A and B
AU2001271614B2 (en) 2000-07-03 2007-05-31 Catalent Pharma Solutions, Llc Host cells containing multiple integrating vectors
WO2002007752A2 (en) 2000-07-24 2002-01-31 Gendel Limited Red blood cell as vehicle for agent-membrane translocation sequence conjugate
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
GB0024550D0 (cg-RX-API-DMAC7.html) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7419829B2 (en) 2000-10-06 2008-09-02 Oxford Biomedica (Uk) Limited Vector system
DK1325138T3 (da) 2000-10-11 2013-10-21 Shire Human Genetic Therapies Optimeret messenger rna
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
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
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
JP4968498B2 (ja) 2002-01-23 2012-07-04 ユニバーシティ オブ ユタ リサーチ ファウンデーション ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発
EP2368982A3 (en) 2002-03-21 2011-10-12 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
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
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
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
CA2562193A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
MX2007013757A (es) 2005-05-05 2008-01-24 Univ Arizona Reagrupacion permitida de secuencia (seer)-met odo de novedad para visualizar secuencias de adn especificas.
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
ES2533370T3 (es) 2005-10-18 2015-04-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
JP4368925B2 (ja) * 2005-11-18 2009-11-18 財団法人 東京都医学研究機構 基質特異性を変換した新規高機能酵素
ATE462783T1 (de) 2006-05-25 2010-04-15 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
EP2765195A1 (en) 2006-05-25 2014-08-13 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
WO2008157688A2 (en) 2007-06-19 2008-12-24 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
HUE042882T2 (hu) * 2008-02-12 2019-07-29 Amicus Therapeutics Inc Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
DE102008025277A1 (de) 2008-05-27 2009-12-03 Merck Patent Gmbh Glaszusammensetzung
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
WO2010065123A1 (en) 2008-12-04 2010-06-10 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP3165234B1 (de) 2009-07-31 2019-04-03 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP2615106B1 (en) 2010-02-08 2018-04-25 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
AU2011215557B2 (en) 2010-02-09 2016-03-10 Sangamo Therapeutics, Inc. Targeted genomic modification with partially single-stranded donor molecules
JP5437895B2 (ja) 2010-04-20 2014-03-12 株式会社ジャパンディスプレイ 表示装置及びその製造方法
WO2011139336A1 (en) 2010-04-26 2011-11-10 Sangamo Biosciences, Inc. Genome editing of a rosa locus using nucleases
KR101974036B1 (ko) 2010-05-03 2019-04-30 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 모듈을 연결하기 위한 조성물
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
US8945868B2 (en) 2010-07-21 2015-02-03 Sangamo Biosciences, Inc. Methods and compositions for modification of a HLA locus
IL293650B2 (en) 2011-09-21 2024-10-01 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
WO2013074999A1 (en) 2011-11-16 2013-05-23 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
KR102166824B1 (ko) 2011-12-22 2020-10-19 에프. 호프만-라 로슈 아게 발현 벡터 구성원 조합, 신규한 제조 세포 생성 방법 및 폴리펩티드의 재조합적 제조를 위한 그의 용도
US20140001721A1 (en) 2012-06-28 2014-01-02 John C. Benko Ergonomic cart
CA2886684C (en) 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
EP2929017A4 (en) 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
AU2014262867B2 (en) 2013-05-10 2019-12-05 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
TW202332774A (zh) 2013-10-23 2023-08-16 美商健臻公司 重組醣蛋白及其用途
DK3080274T3 (da) * 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
BR112016025849A2 (pt) 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
WO2016011448A2 (en) 2014-07-18 2016-01-21 Celltheon Corporation Methods and compositions for expression of polypeptides in a cell
WO2016014794A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
SG10202100131WA (en) 2015-06-23 2021-02-25 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
AU2016343803B2 (en) 2015-10-28 2021-04-29 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA3011049A1 (en) 2016-02-02 2017-08-10 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
BR112019003327A2 (pt) 2016-08-24 2019-07-02 Sangamo Therapeutics, Inc. nucleases manipuladas de alvo específico
EP3528852B1 (en) 2016-10-20 2025-09-03 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
WO2018107026A1 (en) 2016-12-09 2018-06-14 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
EP3568152A1 (en) 2017-01-10 2019-11-20 Amicus Therapeutics, Inc. Recombinant alpha-galactosidase a for treatment of fabry disease
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2019074292A2 (ko) 2017-10-11 2019-04-18 (주)셀트리온 고발현 및 고기능성 목적 단백질의 생산을 위한 발현 카세트 및 이의 이용
CA3089587A1 (en) 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
CA3115944A1 (en) 2018-10-10 2020-04-16 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
MX2021008131A (es) * 2019-01-04 2021-10-13 Sangamo Therapeutics Inc Metodos y composiciones para el tratamiento de la enfermedad de fabry.

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140112896A1 (en) * 2012-07-11 2014-04-24 Sangqmo BioSciences, Inc. Methods and compositions for the provision of proteins deficient in lysosomal storage diseases
US20160060656A1 (en) * 2012-07-11 2016-03-03 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
CHOI JIN-OK; LEE MI HEE; PARK HAE-YOUNG; JUNG SUNG-CHUL:, JOURNAL OF BIOMEDICAL SCIENCE, vol. 17, no. 1, 26, 16 April 2010 (2010-04-16), pages 1 - 10, XP021071211, ISSN: 1423-0127, DOI: 10.1186/1423-0127-17-26 *
JUNG ET AL.: "Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice", PROC NATL ACAD SCI USA, vol. 98, no. 5, 2001, pages 2676 - 81, XP055472677 *
R. SHARMA ET AL: BLOOD, vol. 126, no. 15, 8 October 2015 (2015-10-08), pages 1777 - 1784, XP055354026, ISSN: 0006-4971, DOI: 10.1182/blood-2014-12-615492 *

Also Published As

Publication number Publication date
MX2019004487A (es) 2020-02-07
EP3528852B1 (en) 2025-09-03
BR112019007210A2 (pt) 2019-08-13
RU2019115118A3 (cg-RX-API-DMAC7.html) 2021-06-29
KR20190060829A (ko) 2019-06-03
JP2022078282A (ja) 2022-05-24
AU2017345430A1 (en) 2019-05-02
EP3528852A4 (en) 2020-06-03
US20220273819A1 (en) 2022-09-01
CA3039673A1 (en) 2018-04-26
IL266047B1 (en) 2024-10-01
WO2018075736A1 (en) 2018-04-26
JP7418485B2 (ja) 2024-01-19
JP7042263B2 (ja) 2022-03-25
EP3528852A1 (en) 2019-08-28
US20180117181A1 (en) 2018-05-03
ES3047063T3 (en) 2025-12-03
EP4667565A2 (en) 2025-12-24
IL266047A (en) 2019-06-30
EP3528852C0 (en) 2025-09-03
KR102712926B1 (ko) 2024-10-07
IL266047B2 (en) 2025-02-01
RU2019115118A (ru) 2020-11-20
CN110022904A (zh) 2019-07-16
JP2020500162A (ja) 2020-01-09
US11219695B2 (en) 2022-01-11
CN110022904B (zh) 2024-04-19

Similar Documents

Publication Publication Date Title
US20220273819A1 (en) Methods and compositions for the treatment of fabry disease
US11040115B2 (en) Method for the treatment of lysosomal storage diseases
EP3102673B1 (en) Methods and compositions for treatment of a beta thalessemia
US20200239911A1 (en) Methods and compositions for the treatment of fabry disease
RU2788133C2 (ru) Способы и композиции для лечения болезни фабри
NZ793215A (en) Method And Compositions For The Treatment Of Fabry Disease
HK40011849A (en) Methods and compositions for the treatment of fabry disease
HK40002981B (en) Methods and compositions for the treatment of lysosomal storage diseases
HK40002981A (en) Methods and compositions for the treatment of lysosomal storage diseases
HK1238680B (en) Methods and compositions for the treatment of monogenic diseases

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)