IL259842B - Targeted peptides for the targeting of adeno-associated viruses -aavs- - Google Patents

Targeted peptides for the targeting of adeno-associated viruses -aavs-

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Publication number
IL259842B
IL259842B IL259842A IL25984218A IL259842B IL 259842 B IL259842 B IL 259842B IL 259842 A IL259842 A IL 259842A IL 25984218 A IL25984218 A IL 25984218A IL 259842 B IL259842 B IL 259842B
Authority
IL
Israel
Prior art keywords
aav
seq
sequence
amino acids
php
Prior art date
Application number
IL259842A
Other languages
English (en)
Hebrew (he)
Other versions
IL259842A (en
Inventor
Benjamin E Deverman
Viviana Gradinaru
Ken Y Chan
Original Assignee
California Inst Of Techn
Benjamin E Deverman
Viviana Gradinaru
Ken Y Chan
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 California Inst Of Techn, Benjamin E Deverman, Viviana Gradinaru, Ken Y Chan filed Critical California Inst Of Techn
Publication of IL259842A publication Critical patent/IL259842A/en
Publication of IL259842B publication Critical patent/IL259842B/en

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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/86—Viral vectors
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Families Citing this family (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP7618386B2 (ja) 2017-01-30 2025-01-21 ブレインヴェクティス ポリグルタミン反復脊髄小脳失調症の処置におけるコレステロール２４－ヒドロキシラーゼ（ｈｙｄｒｏｌａｓｅ）のための発現ベクター
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) * 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2018320849A1 (en) * 2017-08-25 2020-03-05 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US12043848B2 (en) * 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3704271B1 (en) 2017-11-02 2026-01-28 California Institute of Technology Neurokinin antagonists and uses thereof
WO2019090000A1 (en) 2017-11-02 2019-05-09 California Institute Of Technology Expression of neuropeptides
US20190202866A1 (en) * 2017-12-22 2019-07-04 William Marsh Rice University Viral nanoparticles and methods of use thereof
EP3762040A4 (en) 2018-03-05 2022-01-05 Children's Medical Center Corporation COMPOSITIONS AND METHODS OF ADMINISTRATION OF NUCLEIC ACIDS TO COCHLEAR AND VESTIBULAR CELLS
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
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
CA3096407A1 (en) * 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
US20210154270A1 (en) * 2018-04-12 2021-05-27 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Anti-aging compositions and methods of use
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2020014471A1 (en) * 2018-07-11 2020-01-16 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
EP3856762A1 (en) 2018-09-28 2021-08-04 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
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020076614A1 (en) 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
US20220054597A1 (en) 2018-10-29 2022-02-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Expression vector for cholesterol 24-hydrolase in therapy of amyotrophic lateral sclerosis
JP2022513618A (ja) * 2018-11-20 2022-02-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 血液脳関門の標的化に有用な組成物および方法
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
US20220143214A1 (en) * 2019-01-30 2022-05-12 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (aavs) for targeted delivery
WO2020160508A1 (en) * 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
US20220249703A1 (en) * 2019-02-15 2022-08-11 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
KR102934700B1 (ko) * 2019-03-28 2026-03-09 더 제너럴 하스피탈 코포레이션 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터
JP2022526018A (ja) * 2019-04-11 2022-05-20 カリフォルニア インスティテュート オブ テクノロジー 脳内への特異性が増強されたウイルス組成物
GB201905301D0 (en) 2019-04-15 2019-05-29 Ospedale San Raffaele Srl Gene therapy
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
JP7585233B2 (ja) * 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ポンペ病の治療のために有用な組成物
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
JP7624410B2 (ja) * 2019-05-21 2025-01-30 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル レット症候群の治療におけるコレステロール ２４－ヒドロラーゼのための発現ベクター
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115087738A (zh) * 2019-08-29 2022-09-20 小利兰·斯坦福大学托管委员会 用于穿越人血脑屏障的腺相关病毒载体
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
GB201912863D0 (en) * 2019-09-06 2019-10-23 Babraham Inst Novel method
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
CN114364436A (zh) * 2019-10-17 2022-04-15 宾州研究基金会 再生功能神经元以用于治疗脊髓损伤和als
US12611436B2 (en) * 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스
KR20220116459A (ko) * 2019-11-22 2022-08-23 더 칠드런스 호스피탈 오브 필라델피아 아데노(adeno)-관련 바이러스 벡터 변이체
CN115279400B (zh) 2020-01-10 2026-04-28 布里格姆妇女医院 用于跨血脑屏障递送免疫治疗剂以治疗脑癌的方法和组合物
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
EP4103214B1 (en) 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
US12552838B2 (en) 2020-04-20 2026-02-17 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US12612627B2 (en) * 2020-04-20 2026-04-28 Regents Of The University Of Minnesota Compositions and methods for increasing sodium current in cardiac cells
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
US20230173102A1 (en) * 2020-04-28 2023-06-08 Genethon Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
AU2021265832A1 (en) 2020-04-29 2022-11-24 The Broad Institute, Inc. Machine learning accelerated protein engineering through fitness prediction
WO2021222831A2 (en) * 2020-05-01 2021-11-04 The Broad Institute, Inc. Engineered central nervous system compositions
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
GB202011871D0 (en) * 2020-07-30 2020-09-16 Cambridge Entpr Ltd Composition and method
KR20230042468A (ko) 2020-08-05 2023-03-28 스페이스크래프트 세븐, 엘엘씨 Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN116406305A (zh) 2020-08-17 2023-07-07 麻省理工学院 Shank3基因治疗方法
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
AU2021329527A1 (en) * 2020-08-21 2023-03-23 Capsida, Inc. Adeno-associated virus compositions having 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
EP4213891A4 (en) * 2020-09-21 2025-07-23 Askbio Inc METHODS OF TREATING NEUROLOGICAL DISEASE
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
CN113754727B (zh) * 2020-09-30 2022-07-12 广州派真生物技术有限公司 腺相关病毒突变体及其应用
EP4229204A1 (en) 2020-10-15 2023-08-23 F. Hoffmann-La Roche AG Nucleic acid constructs for simultaneous gene activation
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022206739A1 (zh) * 2021-03-29 2022-10-06 南京大学 一种基于病毒载体的rna递送系统及其应用
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
WO2022221529A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
KR20240032971A (ko) 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
EP4405373A4 (en) * 2021-09-21 2026-02-11 California Inst Of Techn VIRAL COMPOUNDS TO TARGET THE NERVOUS SYSTEM AND THE LUNGS
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023073526A1 (en) 2021-10-25 2023-05-04 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
CN114107231B (zh) * 2021-12-13 2023-08-18 重庆大学 实现全脑突触后神经元胞体标记的重组腺相关病毒及其应用
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
EP4482970A1 (en) 2022-02-23 2025-01-01 Massachusetts Institute of Technology Methods for upregulating shank3 expression
JP2025514794A (ja) * 2022-04-22 2025-05-09 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア ヒト上衣特異的プロモーターおよびその使用方法
WO2023240158A2 (en) * 2022-06-07 2023-12-14 Oregon Health & Science University Mutant adeno-associated virus (aav) capsids
WO2023240236A1 (en) * 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
US20260085328A1 (en) 2022-09-08 2026-03-26 Voyager Therapeutics, Inc. Controlled expression of viral proteins
GB2622629A (en) 2022-09-23 2024-03-27 Sania Rx Ltd Method
WO2024100145A1 (en) 2022-11-08 2024-05-16 Ospedale San Raffaele S.R.L. Polynucleotide and vector
EP4669330A1 (en) 2023-02-21 2025-12-31 Institut National de la Santé et de la Recherche Médicale METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT OF SANFILIPPO SYNDROME TYPE IIIB
WO2024186275A1 (en) * 2023-03-09 2024-09-12 Agency For Science, Technology And Research Novel compositions of adeno-associated viruses engineered for enhanced tissue transduction and specificity
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN121219020A (zh) 2023-03-22 2025-12-26 圣拉斐尔医院有限责任公司 基因疗法
EP4688807A1 (en) * 2023-03-31 2026-02-11 Exegenesis Bio Singapore Pte. Ltd. Variant aav9 capsid proteins and uses thereof
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4705484A1 (en) * 2023-05-03 2026-03-11 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
TW202444917A (zh) * 2023-05-04 2024-11-16 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
WO2025021839A1 (en) 2023-07-25 2025-01-30 Institut National de la Santé et de la Recherche Médicale Method to treat metabolic disorders
WO2025032158A1 (en) 2023-08-08 2025-02-13 Institut National de la Santé et de la Recherche Médicale Method to treat tauopathies
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN117264912A (zh) * 2023-09-25 2023-12-22 中国科学院深圳先进技术研究院 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用
WO2025073890A1 (en) 2023-10-06 2025-04-10 Institut National de la Santé et de la Recherche Médicale Method to capture circulating tumor extracellular vesicles
GB202316264D0 (en) 2023-10-24 2023-12-06 Ospendale San Raffaele S R L Gene therapy
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
US20250152744A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating neurodegenerative disorders
WO2025157848A1 (en) 2024-01-23 2025-07-31 Institut National de la Santé et de la Recherche Médicale Methods for the intravenous treatment of sanfilippo syndrome type iiib
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
WO2025186293A1 (en) 2024-03-06 2025-09-12 Institut National de la Santé et de la Recherche Médicale Chmp2b mutants for use for treating infection
GB202406331D0 (en) 2024-05-07 2024-06-19 Sania Rx Ltd Capsid
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090202490A1 (en) * 2003-06-30 2009-08-13 Schaffer David V Mutant adeno-associated virus virions and methods of use thereof
US20130142764A1 (en) * 2007-07-14 2013-06-06 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
JP2014518614A (ja) * 2011-04-22 2014-08-07 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 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
US20150079038A1 (en) * 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
US20150315612A1 (en) * 2003-09-30 2015-11-05 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor

Family Cites Families (27)

* 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
US7279313B2 (en) 1995-09-15 2007-10-09 Centelion Circular DNA molecule having a conditional origin of replication, process for their preparation and their use in gene therapy
AU2319497A (en) 1996-03-07 1997-09-22 Regents Of The University Of California, The Helper-free, totally defective adenovirus for gene therapy
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
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
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US6410271B1 (en) 2000-06-23 2002-06-25 Genetastix Corporation Generation of highly diverse library of expression vectors via homologous recombination in yeast
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
WO2006099615A2 (en) 2005-03-16 2006-09-21 The Johns Hopkins University Adenoviral fiber exchange shuttle system
CN103849629B (zh) * 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
EP2132309A4 (en) 2007-02-23 2011-01-05 Univ Florida COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES
US9018138B2 (en) 2007-08-16 2015-04-28 The Johns Hopkins University Compositions and methods for generating and screening adenoviral libraries
US20110223635A1 (en) 2008-08-11 2011-09-15 Karl Deisseroth Method and Composition for Controlling Gene Expression
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
JP6013654B2 (ja) 2013-09-19 2016-10-25 クルセル ホランド ベー ヴェー 改良されたアデノウイルス製剤
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
EP3619310A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090202490A1 (en) * 2003-06-30 2009-08-13 Schaffer David V Mutant adeno-associated virus virions and methods of use thereof
US20150315612A1 (en) * 2003-09-30 2015-11-05 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20130142764A1 (en) * 2007-07-14 2013-06-06 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
JP2014518614A (ja) * 2011-04-22 2014-08-07 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 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
US20150079038A1 (en) * 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery

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