MX2021000443A - Vectores de terapia génica para el tratamiento de la enfermedad de danon. - Google Patents

Vectores de terapia génica para el tratamiento de la enfermedad de danon.

Info

Publication number
MX2021000443A
MX2021000443A MX2021000443A MX2021000443A MX2021000443A MX 2021000443 A MX2021000443 A MX 2021000443A MX 2021000443 A MX2021000443 A MX 2021000443A MX 2021000443 A MX2021000443 A MX 2021000443A MX 2021000443 A MX2021000443 A MX 2021000443A
Authority
MX
Mexico
Prior art keywords
gene therapy
danon
disease
treatment
lamp
Prior art date
Application number
MX2021000443A
Other languages
English (en)
Spanish (es)
Inventor
Annahita Keravala
Simon Moore
David Ricks
Original Assignee
Spacecraft Seven Llc
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 Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of MX2021000443A publication Critical patent/MX2021000443A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/04—Uses of viruses as vector in vivo
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cardiology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
MX2021000443A 2018-07-12 2019-07-11 Vectores de terapia génica para el tratamiento de la enfermedad de danon. MX2021000443A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862697302P 2018-07-12 2018-07-12
PCT/US2019/041465 WO2020014523A1 (en) 2018-07-12 2019-07-11 Gene therapy vectors for treatment of danon disease

Publications (1)

Publication Number Publication Date
MX2021000443A true MX2021000443A (es) 2021-05-28

Family

ID=69141704

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021000443A MX2021000443A (es) 2018-07-12 2019-07-11 Vectores de terapia génica para el tratamiento de la enfermedad de danon.

Country Status (12)

Country Link
US (2) US10703797B2 (enExample)
EP (1) EP3820537A4 (enExample)
JP (2) JP7586810B2 (enExample)
KR (1) KR102918544B1 (enExample)
CN (1) CN112512596B (enExample)
AU (1) AU2019299970B2 (enExample)
BR (1) BR112021000509A2 (enExample)
CA (1) CA3106010A1 (enExample)
IL (1) IL279918A (enExample)
MX (1) MX2021000443A (enExample)
SG (1) SG11202100022SA (enExample)
WO (1) WO2020014523A1 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
IL280465B2 (en) 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
US20220143215A1 (en) * 2019-02-12 2022-05-12 Spacecraft Seven, Llc Gene therapy vectors for treatment of danon disease
WO2021142267A1 (en) 2020-01-08 2021-07-15 Scarpmap Llc Methods and computing system for processing ultrasound image to determine health of subdermal tissue
WO2022017630A1 (en) * 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
WO2022026516A1 (en) * 2020-07-29 2022-02-03 The Board Of Regents Of The University Of Texas System Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
EP4217010A4 (en) * 2020-09-28 2025-01-08 Tacit Therapeutics, Inc. TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES
JP2023548746A (ja) * 2020-10-13 2023-11-21 クリヤ セラピューティクス インコーポレイテッド サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
MX2023005901A (es) * 2020-11-20 2023-08-29 Baylor College Medicine Supresión de señalización hippo en el nicho de células madre promueve la regeneración de músculo esquelético.
CN116887868A (zh) * 2020-12-07 2023-10-13 太空飞船七有限责任公司 达农病的治疗
BR112023021037A2 (pt) * 2021-04-12 2023-12-19 Univ California Terapia gênica para cardiomiopatia ventricular direita arritmogênica
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣
JP2024533163A (ja) 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集
KR102577502B1 (ko) * 2021-09-08 2023-09-13 주식회사 꿈랩 생체분자의 안정적 발현 및 전달을 위한 플라스미드 플랫폼
WO2023038479A1 (ko) * 2021-09-08 2023-03-16 주식회사 꿈랩 생체분자의 안정적 발현 및 전달을 위한 플라스미드 플랫폼
WO2025217543A1 (en) * 2024-04-12 2025-10-16 AskBio Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding lamp2b

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
ATE237694T1 (de) 1991-08-20 2003-05-15 Us Gov Health & Human Serv Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
JP3866760B2 (ja) 1993-02-22 2007-01-10 ザ ロックフェラー ユニバーシティ 一過性トランスフェクションによる高力価のヘルパーフリーのレトロウイルスの生産
FR2712812B1 (fr) 1993-11-23 1996-02-09 Centre Nat Rech Scient Composition pour la production de produits thérapeutiques in vivo.
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US20020065236A1 (en) * 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーｒｎａのスプライス部位選択の再プログラミング方法
AU2003295881A1 (en) * 2002-11-25 2004-06-18 Exelixis, Inc. Lamps as modifiers of the p53 pathway and methods of use
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CA2641070C (en) 2006-02-06 2017-01-03 Ramot At Tel-Aviv University Ltd. Enzyme replacement therapy for treating lysosomal storage diseases
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
PL2158322T3 (pl) 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
CA2799944A1 (en) * 2010-05-20 2011-11-24 University Of Rochester Methods and compositions related to modulating autophagy
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US10648001B2 (en) * 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
AU2013332061A1 (en) 2012-10-17 2015-05-07 Fondazione Telethon Gene therapy for glycogen storage diseases
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
EP3405215B1 (en) 2016-01-19 2022-06-15 The Regents of the University of California Methods for the treatment of danon disease and other disorders of autophagy
IL320371A (en) * 2016-09-08 2025-06-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Gene therapy for patients with fanconi anemia
EP3519569B1 (en) 2016-09-30 2020-12-02 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
AU2018236353B2 (en) 2017-03-15 2023-12-21 The Regents Of The University Of California Methods of treating lysosomal disorders
US20220143215A1 (en) * 2019-02-12 2022-05-12 Spacecraft Seven, Llc Gene therapy vectors for treatment of danon disease

Also Published As

Publication number Publication date
US20200148745A1 (en) 2020-05-14
JP2024150588A (ja) 2024-10-23
CN112512596A (zh) 2021-03-16
JP2021531752A (ja) 2021-11-25
EP3820537A4 (en) 2022-04-13
AU2019299970B2 (en) 2025-12-11
CA3106010A1 (en) 2020-01-16
EP3820537A1 (en) 2021-05-19
KR102918544B1 (ko) 2026-01-27
IL279918A (en) 2021-03-01
WO2020014523A1 (en) 2020-01-16
US10703797B2 (en) 2020-07-07
CN112512596B (zh) 2025-12-12
BR112021000509A2 (pt) 2021-04-06
AU2019299970A1 (en) 2021-01-28
KR20210030965A (ko) 2021-03-18
US20210381003A1 (en) 2021-12-09
SG11202100022SA (en) 2021-01-28
JP7586810B2 (ja) 2024-11-19

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