JP7624724B2 - 標的遺伝子療法のためのアデノ随伴ウイルス組成物 - Google Patents

標的遺伝子療法のためのアデノ随伴ウイルス組成物 Download PDF

Info

Publication number
JP7624724B2
JP7624724B2 JP2021517037A JP2021517037A JP7624724B2 JP 7624724 B2 JP7624724 B2 JP 7624724B2 JP 2021517037 A JP2021517037 A JP 2021517037A JP 2021517037 A JP2021517037 A JP 2021517037A JP 7624724 B2 JP7624724 B2 JP 7624724B2
Authority
JP
Japan
Prior art keywords
amino acid
seq
aav capsid
variant
enrichment
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
JP2021517037A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022502046A (ja
JP2022502046A5 (enExample
Inventor
ヴィヴィアナ グラディナル，
ニコラス シー． フリトザニス，
ニコラス エス． ゴーデン，
Original Assignee
カリフォルニア インスティテュート オブ テクノロジー
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 カリフォルニア インスティテュート オブ テクノロジー filed Critical カリフォルニア インスティテュート オブ テクノロジー
Publication of JP2022502046A publication Critical patent/JP2022502046A/ja
Publication of JP2022502046A5 publication Critical patent/JP2022502046A5/ja
Priority to JP2024134976A priority Critical patent/JP2024161454A/ja
Application granted granted Critical
Publication of JP7624724B2 publication Critical patent/JP7624724B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • 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
    • C12N2750/14071—Demonstrated in vivo effect
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021517037A 2018-09-26 2019-09-25 標的遺伝子療法のためのアデノ随伴ウイルス組成物 Active JP7624724B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024134976A JP2024161454A (ja) 2018-09-26 2024-08-13 標的遺伝子療法のためのアデノ随伴ウイルス組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862736904P 2018-09-26 2018-09-26
US62/736,904 2018-09-26
US201962832812P 2019-04-11 2019-04-11
US62/832,812 2019-04-11
PCT/US2019/052969 WO2020068990A1 (en) 2018-09-26 2019-09-25 Adeno-associated virus compositions for targeted gene therapy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024134976A Division JP2024161454A (ja) 2018-09-26 2024-08-13 標的遺伝子療法のためのアデノ随伴ウイルス組成物

Publications (3)

Publication Number Publication Date
JP2022502046A JP2022502046A (ja) 2022-01-11
JP2022502046A5 JP2022502046A5 (enExample) 2022-10-03
JP7624724B2 true JP7624724B2 (ja) 2025-01-31

Family

ID=69953332

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021517037A Active JP7624724B2 (ja) 2018-09-26 2019-09-25 標的遺伝子療法のためのアデノ随伴ウイルス組成物
JP2024134976A Pending JP2024161454A (ja) 2018-09-26 2024-08-13 標的遺伝子療法のためのアデノ随伴ウイルス組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024134976A Pending JP2024161454A (ja) 2018-09-26 2024-08-13 標的遺伝子療法のためのアデノ随伴ウイルス組成物

Country Status (9)

Country Link
US (3) US11149256B2 (enExample)
EP (1) EP3856913A4 (enExample)
JP (2) JP7624724B2 (enExample)
KR (1) KR20210084459A (enExample)
CN (1) CN113966399A (enExample)
AU (1) AU2019346447A1 (enExample)
CA (1) CA3114199A1 (enExample)
SG (1) SG11202103102WA (enExample)
WO (1) WO2020068990A1 (enExample)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020533968A (ja) * 2017-08-25 2020-11-26 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 組換えアデノ随伴ベクター
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
JP2021530218A (ja) 2018-07-11 2021-11-11 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 血液脳関門を超える薬剤の送達のための方法および組成物
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
AU2020214831B2 (en) * 2019-01-30 2025-09-25 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (AAVs) for targeted delivery
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP4061947A4 (en) 2019-11-22 2024-04-24 California Institute of Technology METHODS FOR ROBUST CONTROL OF GENE EXPRESSION
CA3167290A1 (en) 2020-01-10 2021-07-15 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
WO2021222232A1 (en) * 2020-04-28 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
AU2021265832A1 (en) 2020-04-29 2022-11-24 The Broad Institute, Inc. Machine learning accelerated protein engineering through fitness prediction
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
WO2022040530A2 (en) * 2020-08-21 2022-02-24 Capsida, Inc. Virus compositions for treating mucopolysaccharidosis ii
EP4199972A4 (en) * 2020-08-21 2025-01-15 Capsida, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS WITH PREFERRED EXPRESSION LEVELS
US12129287B2 (en) * 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
US20240226331A9 (en) * 2021-02-22 2024-07-11 Decibel Therapeutics, Inc. Methods and compositions for reducing nucleic acid vector-induced toxicity in the inner ear
US20250281643A1 (en) 2021-04-13 2025-09-11 Capsida, Inc. Selected aav compositions having preferred brain enrichment
US20240299586A1 (en) * 2021-05-06 2024-09-12 The Regents Of The University Of California Novel aav serotypes derived from a library screen
WO2022266452A1 (en) * 2021-06-18 2022-12-22 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
KR20240036634A (ko) * 2021-07-23 2024-03-20 레터럴 아이피 피티와이 리미티드 란티오닌 합성효소 c-유사 단백질(lancl)에 결합할 수 있는 펩티드 조성물 및 그의 용도
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
US20240382617A1 (en) * 2021-09-21 2024-11-21 Capsida Biotherapeutics, Inc. Virus compositions to efficiently target the nervous system and the lungs
JP2025500347A (ja) * 2021-10-05 2025-01-09 ザ・ブロード・インスティテュート・インコーポレイテッド 操作された心筋組成物
KR20240095539A (ko) * 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023069744A1 (en) * 2021-10-22 2023-04-27 Temple University Of The Commonwealth System Of Higher Education Bag3 methods and uses for treatment of inflammation
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4433169A1 (en) * 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US11732566B2 (en) 2021-11-22 2023-08-22 Saudi Arabian Oil Company Slickwater hydraulic fracturing with exothermic reactants
EP4440696A1 (en) * 2021-12-01 2024-10-09 Shape Therapeutics Inc. Functional aav capsids for systemic administration
CN114146015B (zh) * 2021-12-16 2023-07-21 大连深蓝肽科技研发有限公司 一种含有花胶低聚肽的祛斑美容组合物及其制备方法和用途
JP2025534229A (ja) * 2022-09-26 2025-10-15 エンコーデド セラピューティクス，インク． 後根神経節及び／又は肝臓における遺伝子発現を脱標的化するための要素
KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
EP4410987A1 (en) * 2023-01-31 2024-08-07 Universitätsklinikum Hamburg-Eppendorf An aav9 capsid variant for targeted gene transfer
EP4410988A1 (en) * 2023-01-31 2024-08-07 Universitätsklinikum Hamburg-Eppendorf An aav2-vector variant for targeted transfer of genes
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
CN116790612B (zh) * 2023-06-21 2024-02-02 江苏省人民医院（南京医科大学第一附属医院） 一种促进心肌再生修复的过氧化物酶体生发蛋白3及其应用
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
CN117286119A (zh) * 2023-09-06 2023-12-26 复旦大学附属中山医院 可静脉注射给药的tpk表达病毒及其治疗阿尔茨海默病的应用
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2026024045A1 (ko) * 2024-07-22 2026-01-29 한국생명공학연구원 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도
WO2026050233A1 (en) * 2024-08-26 2026-03-05 Capsida Biotherapeutics, Inc. Novel adeno-associated virus compositions having increased brain enrichment
CN118754952B (zh) * 2024-09-06 2025-01-10 上海玮美基因科技有限责任公司 一种新型腺相关病毒及其应用
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
US20180230186A1 (en) 2013-09-13 2018-08-16 California Institute Of Technology Selective recovery

Family Cites Families (82)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5919676A (en) 1993-06-24 1999-07-06 Advec, Inc. Adenoviral vector system comprising Cre-loxP recombination
US6379943B1 (en) 1999-03-05 2002-04-30 Merck & Co., Inc. High-efficiency Cre/loxp based system for construction of adenovirus vectors
US6228646B1 (en) 1996-03-07 2001-05-08 The Regents Of The University Of California Helper-free, totally defective adenovirus for gene therapy
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
WO1998010086A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US6429001B1 (en) 2000-01-26 2002-08-06 Chiron Corporation Recombinant AAV packaging systems
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6410271B1 (en) 2000-06-23 2002-06-25 Genetastix Corporation Generation of highly diverse library of expression vectors via homologous recombination in yeast
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
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
WO2006099615A2 (en) 2005-03-16 2006-09-21 The Johns Hopkins University Adenoviral fiber exchange shuttle system
US20090093403A1 (en) 2007-03-01 2009-04-09 Feng Zhang Systems, methods and compositions for optical stimulation of target cells
US7588772B2 (en) * 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
WO2008103993A2 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
CA2693712C (en) 2007-07-14 2018-05-29 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US9018138B2 (en) 2007-08-16 2015-04-28 The Johns Hopkins University Compositions and methods for generating and screening adenoviral libraries
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
ES2701349T3 (es) 2008-04-23 2019-02-21 Univ Leland Stanford Junior Sistemas, métodos y composiciones para la estimulación óptica de células diana
US20110223635A1 (en) 2008-08-11 2011-09-15 Karl Deisseroth Method and Composition for Controlling Gene Expression
EP3399024A1 (en) 2010-03-17 2018-11-07 The Board of Trustees of The Leland Stanford Junior University Light-sensitive ion-passing molecules
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
WO2012103343A1 (en) 2011-01-28 2012-08-02 The Board Of Trustees Of The Leland Stanford Junior University Functional targeted brain endoskeletonization
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
CA2859364C (en) 2011-12-16 2019-05-07 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
ES2628006T3 (es) 2012-02-21 2017-08-01 Circuit Therapeutics, Inc. Composiciones para el tratamiento de trastornos neurogénicos del suelo pélvico
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
JP2016523835A (ja) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
CN111514314A (zh) 2013-09-19 2020-08-11 扬森疫苗与预防公司 改进的腺病毒配制品
US10794802B2 (en) 2013-09-20 2020-10-06 California Institute Of Technology Whole-body tissue stabilization and selective extractions via tissue-hydrogel hybrids for high resolution intact circuit mapping and phenotyping
US9778154B2 (en) 2013-09-20 2017-10-03 California Institute Of Technology Methods for phenotyping of intact whole tissues
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
AU2016343979A1 (en) 2015-10-29 2018-05-10 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
HRP20231451T1 (hr) 2016-02-05 2024-03-01 Emory University Ubrizgavanje jednolančanog ili samo-komplementarnog adeno-povezanog virusa 9 u cerebrospinalnu tekućinu
CA3014637A1 (en) 2016-02-16 2017-08-24 Mark A. Kay Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CN109640949B (zh) 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
US9927975B2 (en) 2016-08-03 2018-03-27 Micron Technology, Inc. Hybrid memory drives, computer system, and related method for operating a multi-mode hybrid drive
WO2018045347A1 (en) 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
US20190269797A1 (en) 2016-11-04 2019-09-05 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
US20230048025A1 (en) 2016-12-22 2023-02-16 Oregon Health & Science University Adeno associated viral vectors
EP3583220A4 (en) 2017-02-15 2021-07-07 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM
JP2020510424A (ja) * 2017-02-28 2020-04-09 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 改変ａａｖカプシドおよびその使用
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
CN117821509A (zh) 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
EP3618839A4 (en) * 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2018222503A1 (en) 2017-05-31 2018-12-06 The Regents Of The University Of California Adeno-associated virus with variant capsid and methods of use thereof
KR20250023600A (ko) 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
EP3774853A1 (en) 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
CA3097375A1 (en) 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210207167A1 (en) 2018-05-16 2021-07-08 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
EP3830107A2 (en) 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US11858969B2 (en) 2018-09-18 2024-01-02 California Institute Of Technology Engineered light-sensitive proteins
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP7659271B2 (ja) 2019-03-28 2025-04-09 ザ ジェネラル ホスピタル コーポレイション 導入遺伝子発現のための操作されたアデノ随伴（ａａｖ）ベクター
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
SG11202111249YA (en) 2019-04-11 2021-11-29 California Inst Of Techn Virus compositions with enhanced specificity in the brain
EA202192801A1 (ru) 2019-04-12 2022-02-24 Инкоудид Терапьютикс, Инк. Композиции и способы введения лекарственных средств
CN114269926A (zh) 2019-04-26 2022-04-01 美商圣加莫治疗股份有限公司 Aav工程化
SG11202113048SA (en) 2019-05-29 2021-12-30 Encoded Therapeutics Inc Compositions and methods for selective gene regulation

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180230186A1 (en) 2013-09-13 2018-08-16 California Institute Of Technology Selective recovery
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
From single-cell to whole-body: developing a molecular neuroscience toolkit，Caltech，2020年02月19日，1-90
Molecular Therapy，2018年01月，Vol. 26 No 1，304-319, Supplemental Information
ウイルス，2007年，第５７巻、第１号，ｐｐ．４７－５６

Also Published As

Publication number Publication date
JP2022502046A (ja) 2022-01-11
EP3856913A1 (en) 2021-08-04
US20220119775A1 (en) 2022-04-21
EP3856913A4 (en) 2022-10-26
US11149256B2 (en) 2021-10-19
WO2020068990A1 (en) 2020-04-02
KR20210084459A (ko) 2021-07-07
US20200165576A1 (en) 2020-05-28
US20240101969A9 (en) 2024-03-28
US12049648B2 (en) 2024-07-30
US20250179441A1 (en) 2025-06-05
JP2024161454A (ja) 2024-11-19
CN113966399A (zh) 2022-01-21
SG11202103102WA (en) 2021-04-29
AU2019346447A1 (en) 2021-04-29
CA3114199A1 (en) 2020-04-02

Similar Documents

Publication Publication Date Title
JP2024161454A (ja) 標的遺伝子療法のためのアデノ随伴ウイルス組成物
JP2025106514A (ja) 脳内への特異性が増強されたウイルス組成物
US20250082779A1 (en) Adeno-associated virus compositions having increased brain enrichment
US20240150410A1 (en) Aav compositions having high expression levels in brain
WO2022040527A2 (en) Adeno-associated virus compositions having preferred expression levels
US20250249124A1 (en) Adeno-associated virus compositions having increased brain enrichment
US20260028595A1 (en) Selected adeno-associated virus compositions having preferred brain, spinal cord, and/or heart expression levels
US20250346637A1 (en) Adeno-associated virus compositions having increased brain enrichment and decreased liver enrichment
JP2025509971A (ja) 好ましい脳エンリッチメントを有する選択されたａａｖ組成物
JP2025535374A (ja) 好ましい脳エンリッチメントおよび低い肝臓エンリッチメントを有するアデノ随伴ウイルス組成物
CN121844054A (zh) 具有增加的脑富集的腺相关病毒组合物

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210921

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220922

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220922

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230825

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20230825

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231114

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240119

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240226

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240410

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240813

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20240823

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241127

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241212

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20241225

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250114

R150 Certificate of patent or registration of utility model

Ref document number: 7624724

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150