JP7291397B2 - 血管系を通過する遺伝子移入の方法および組成物 - Google Patents

血管系を通過する遺伝子移入の方法および組成物 Download PDF

Info

Publication number
JP7291397B2
JP7291397B2 JP2019542561A JP2019542561A JP7291397B2 JP 7291397 B2 JP7291397 B2 JP 7291397B2 JP 2019542561 A JP2019542561 A JP 2019542561A JP 2019542561 A JP2019542561 A JP 2019542561A JP 7291397 B2 JP7291397 B2 JP 7291397B2
Authority
JP
Japan
Prior art keywords
aav
amino acid
seq
cells
capsid
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
JP2019542561A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020505936A5 (enExample
JP2020505936A (ja
Inventor
アラヴィンド アソカン
ムルリダラン，ギリダル
ブレイク オルブライト
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.)
University of North Carolina at Chapel Hill
Original Assignee
University of North Carolina at Chapel Hill
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 University of North Carolina at Chapel Hill filed Critical University of North Carolina at Chapel Hill
Publication of JP2020505936A publication Critical patent/JP2020505936A/ja
Publication of JP2020505936A5 publication Critical patent/JP2020505936A5/ja
Priority to JP2023007075A priority Critical patent/JP7574508B2/ja
Application granted granted Critical
Publication of JP7291397B2 publication Critical patent/JP7291397B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • A61P9/10—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
    • 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
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2019542561A 2017-02-15 2018-02-15 血管系を通過する遺伝子移入の方法および組成物 Active JP7291397B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023007075A JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762459286P 2017-02-15 2017-02-15
US62/459,286 2017-02-15
PCT/US2018/018381 WO2018152333A1 (en) 2017-02-15 2018-02-15 Methods and compositions for gene transfer across the vasculature

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023007075A Division JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Publications (3)

Publication Number Publication Date
JP2020505936A JP2020505936A (ja) 2020-02-27
JP2020505936A5 JP2020505936A5 (enExample) 2021-03-04
JP7291397B2 true JP7291397B2 (ja) 2023-06-15

Family

ID=63169716

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019542561A Active JP7291397B2 (ja) 2017-02-15 2018-02-15 血管系を通過する遺伝子移入の方法および組成物
JP2023007075A Active JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023007075A Active JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Country Status (13)

Country Link
US (2) US11905312B2 (enExample)
EP (1) EP3583220A4 (enExample)
JP (2) JP7291397B2 (enExample)
KR (1) KR20190117571A (enExample)
CN (2) CN110520536B (enExample)
AU (1) AU2018221738B2 (enExample)
BR (1) BR112019016769A2 (enExample)
CA (1) CA3052829A1 (enExample)
EA (1) EA038695B1 (enExample)
IL (2) IL312788A (enExample)
MX (1) MX2019009720A (enExample)
SG (1) SG11201907298WA (enExample)
WO (1) WO2018152333A1 (enExample)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
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
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
IL278598B2 (en) 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
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
CN119390787A (zh) * 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
CN113795575A (zh) 2019-02-04 2021-12-14 自由行疗法有限公司 多核苷酸
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
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
US20220195462A1 (en) * 2019-04-24 2022-06-23 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 遺伝子治療のためのａａｖカプシドバリアント
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
US20220411821A1 (en) * 2019-10-28 2022-12-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
CN115023242B (zh) * 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
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
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
TWI910162B (zh) 2020-05-05 2026-01-01 美國公爵大學 跨物種相容腺相關病毒組合物及其使用方法
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
WO2022076750A2 (en) * 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20230374541A1 (en) * 2020-10-07 2023-11-23 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
WO2022150634A2 (en) * 2021-01-08 2022-07-14 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
EP4277524A4 (en) * 2021-01-13 2025-06-04 Duke University Compositions for and methods of improving fluid flux in the brain
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
US20250281639A1 (en) * 2021-07-14 2025-09-11 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating chronic pain and for retrograde transduction of neurons
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CA3229580A1 (en) * 2021-08-20 2023-02-23 Anna Nikolaevna STRELKOVA Method for producing modified adeno-associated virus capsid
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
US20240352077A1 (en) * 2021-08-25 2024-10-24 Children's Medical Research Institute Modified aav capsids and vectors
WO2023023781A1 (en) * 2021-08-25 2023-03-02 Children's Medical Research Institute Modified aav capsid polypeptides and vectors
EP4413019A2 (en) * 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
WO2023201277A1 (en) * 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4695396A1 (en) 2023-04-10 2026-02-18 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
AU2024294136A1 (en) * 2023-07-20 2026-02-05 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
KR102868933B1 (ko) * 2023-11-08 2025-10-14 주식회사 글루진테라퓨틱스 성상세포 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
KR102844372B1 (ko) * 2023-12-06 2025-08-11 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용
AU2025206518A1 (en) * 2024-01-11 2026-07-23 Children's Medical Research Institute Aav capsids and vectors for transduction of cells
CN119405806A (zh) * 2024-10-25 2025-02-11 南通大学 小肌营养蛋白域含2抑制剂在制备促进神经生长药物中的应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1135468B1 (en) * 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
DE19933288A1 (de) * 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
SI1496944T1 (sl) * 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
EP2396343B1 (en) * 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP2782596A4 (en) * 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENIC ADMINISTRATION
PL2900686T3 (pl) * 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
CN111206032B (zh) * 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
WO2017015102A1 (en) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids

Also Published As

Publication number Publication date
EA038695B1 (ru) 2021-10-06
IL268534B2 (en) 2024-10-01
CN110520536B (zh) 2024-07-09
IL268534A (en) 2019-09-26
WO2018152333A9 (en) 2019-01-24
KR20190117571A (ko) 2019-10-16
US20190367562A1 (en) 2019-12-05
US11905312B2 (en) 2024-02-20
JP2023052541A (ja) 2023-04-11
EP3583220A1 (en) 2019-12-25
CA3052829A1 (en) 2018-08-23
CN118978574A (zh) 2024-11-19
IL268534B1 (en) 2024-06-01
AU2018221738B2 (en) 2024-06-27
JP2020505936A (ja) 2020-02-27
EP3583220A4 (en) 2021-07-07
JP7574508B2 (ja) 2024-10-29
WO2018152333A1 (en) 2018-08-23
SG11201907298WA (en) 2019-09-27
BR112019016769A2 (pt) 2020-05-26
US20240209031A1 (en) 2024-06-27
CN110520536A (zh) 2019-11-29
IL312788A (en) 2024-07-01
MX2019009720A (es) 2019-10-07
AU2018221738A1 (en) 2019-08-22
EA201991911A1 (ru) 2020-02-20

Similar Documents

Publication Publication Date Title
JP7574508B2 (ja) 血管系を通過する遺伝子移入の方法および組成物
US12281335B2 (en) Methods and compositions for delivery of viral vectors across the blood-brain barrier
JP6042825B2 (ja) 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
CN110770346B (zh) 多倍体腺相关病毒载体及其制备和使用方法
JP7393766B2 (ja) 抗体回避ウイルスベクターのための方法および組成物
JP2021502086A (ja) 環状ｒｎａ分子のための方法及び組成物
JP2018503376A (ja) 標的化遺伝子移入のための方法および組成物
CN110691846A (zh) 合理的多倍体腺相关病毒载体及其制造和使用方法
EP4277920A1 (en) Aav vectors targeting t-cells
CN118420723B (zh) 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用
WO2024124019A2 (en) Aav vectors targeting hematopoietic stem cells
HK40038424A (en) Methods and compositions for delivery of viral vectors across the blood-brain barrier
HK40022719B (en) Polyploid adeno-associated virus vectors and methods of making and using the same

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210119

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210119

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220303

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220526

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20220921

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230120

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20230120

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20230201

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230203

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

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20230224

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20230227

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230417

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230420

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: 20230508

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230529

R150 Certificate of patent or registration of utility model

Ref document number: 7291397

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150