BR112018011711A2 - peptídeos de direcionamento para direcionar virus adeno-associados (aav) - Google Patents

peptídeos de direcionamento para direcionar virus adeno-associados (aav)

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Publication number
BR112018011711A2
BR112018011711A2 BR112018011711A BR112018011711A BR112018011711A2 BR 112018011711 A2 BR112018011711 A2 BR 112018011711A2 BR 112018011711 A BR112018011711 A BR 112018011711A BR 112018011711 A BR112018011711 A BR 112018011711A BR 112018011711 A2 BR112018011711 A2 BR 112018011711A2
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BR
Brazil
Prior art keywords
aav
adeno
associated virus
target
targeting peptides
Prior art date
Application number
BR112018011711A
Other languages
English (en)
Inventor
E Deverman Benjamin
Y Chan Ken
Gradinaru Viviana
Original Assignee
California Inst Of Techn
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 filed Critical California Inst Of Techn
Publication of BR112018011711A2 publication Critical patent/BR112018011711A2/pt

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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/02—Peptides of undefined number of amino acids; Derivatives thereof
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/08—Peptides having 5 to 11 amino acids
    • A—HUMAN NECESSITIES
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    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/2093—Leukaemia inhibitory factor [LIF]
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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Abstract

trata-se de sequências peptídicas com capacidade para direcionar vírus adenoassociados (aav) para ambientes específicos alvo, por exemplo, o sistema nervoso e o coração, em um indivíduo. também são divulgados os aavs que têm proteínas capsidiais que não ocorrem naturalmente que compreendem as sequências peptídicas divulgadas, e métodos de uso dos aavs para tratar doenças.
BR112018011711A 2015-12-11 2016-12-09 peptídeos de direcionamento para direcionar virus adeno-associados (aav) BR112018011711A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562266184P 2015-12-11 2015-12-11
US201662421891P 2016-11-14 2016-11-14
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Families Citing this family (125)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
RU2749882C2 (ru) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
AU2015364636B9 (en) * 2014-12-16 2021-12-02 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
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
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
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California METHOD FOR BIOMEDICAL TARGETING AND RELEASE, AND DEVICES AND SYSTEMS FOR IMPLEMENTING THEM
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
EP3662060A2 (en) * 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
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.
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US11376337B2 (en) 2017-11-02 2022-07-05 California Institute Of Technology Expression of neuropeptides
WO2019090001A1 (en) 2017-11-02 2019-05-09 California Institute Of Technology Neurokinin antagonists and uses thereof
US20190202866A1 (en) * 2017-12-22 2019-07-04 William Marsh Rice University Viral nanoparticles and methods of use thereof
JP7378417B2 (ja) * 2018-03-30 2023-11-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 増強されたヒト膵臓トロピズムを有する新規な組換えアデノ随伴ウイルスキャプシド
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
AU2019253700A1 (en) * 2018-04-09 2020-11-26 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
EP3773677A1 (en) * 2018-04-12 2021-02-17 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Anti-aging compositions and methods of use
MA52631A (fr) 2018-05-15 2021-03-24 Voyager Therapeutics Inc Compositions et méthodes pour le traitement de la maladie de parkinson
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CN112703198A (zh) 2018-07-11 2021-04-23 布里格姆妇女医院 用于跨血脑屏障递送试剂的方法和组合物
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
EP3830107A2 (en) 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020068990A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2020069461A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020072683A1 (en) 2018-10-02 2020-04-09 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
JP2022512621A (ja) 2018-10-05 2022-02-07 ボイジャー セラピューティクス インコーポレイテッド Ａａｖ産生タンパク質をコードする操作された核酸コンストラクト
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3116701A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
HUE060949T2 (hu) 2018-10-29 2023-04-28 Inst Nat Sante Rech Med Koleszterin 24-hidroláz expressziós vektor amiotrófiás laterálszklerózis kezelésében
CN113395975A (zh) * 2018-11-20 2021-09-14 宾夕法尼亚州大学信托人 用于靶向血脑屏障的组合物和方法
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
CA3128205A1 (en) * 2019-01-30 2020-08-06 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (aavs) for targeted delivery
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
AU2020223367A1 (en) * 2019-02-15 2021-10-07 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
KR20210143869A (ko) * 2019-03-28 2021-11-29 더 제너럴 하스피탈 코포레이션 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터
WO2020210655A1 (en) * 2019-04-11 2020-10-15 California Institute Of Technology Virus compositions with enhanced specificity in the brain
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
JP2022530824A (ja) * 2019-04-30 2022-07-01 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
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
WO2020234363A2 (en) 2019-05-21 2020-11-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome
US20220281922A1 (en) 2019-08-02 2022-09-08 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
US20220364114A1 (en) 2019-08-26 2022-11-17 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
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
US20210162002A1 (en) * 2019-10-17 2021-06-03 The Penn State Research Foundation Regenerating functional neurons for treatment of spinal cord injury and als
CN114585743A (zh) * 2019-10-17 2022-06-03 斯特里迪比奥公司 Aav转移盒
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
IL293068A (en) * 2019-11-19 2022-07-01 Asklepios Biopharmaceutical Inc A therapeutic adeno-associated virus containing liver-specific promoters for the treatment of Pompe disease and lysosomal disorders
US11981705B2 (en) 2020-01-10 2024-05-14 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
AU2021218411A1 (en) 2020-02-13 2022-10-06 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
CA3180202A1 (en) * 2020-04-20 2021-10-28 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
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
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
BR112022023106A2 (pt) * 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
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
CN117120619A (zh) 2020-07-27 2023-11-24 沃雅戈治疗公司 用于治疗与葡萄糖神经酰胺酶β缺陷相关的神经病症的组合物和方法
EP4192962A1 (en) 2020-08-05 2023-06-14 Spacecraft Seven, LLC Csrp3 (cysteine and glycine rich protein 3) gene therapy
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
US20230340041A1 (en) 2020-08-17 2023-10-26 Massachusetts Institute Of Technology Shank3 gene therapy approaches
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
WO2022040527A2 (en) * 2020-08-21 2022-02-24 Capsida, Inc. Adeno-associated virus compositions having preferred expression levels
US20230365963A1 (en) * 2020-09-21 2023-11-16 Asklepios Biopharmaceutical, Inc. Methods for treating neurological disease
CN113717248B (zh) * 2020-09-30 2022-07-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
US20220154223A1 (en) 2020-10-15 2022-05-19 Hoffmann-La Roche Inc. Nucleic acid constructs for simultaneous gene activation
CN116648448A (zh) 2020-11-11 2023-08-25 欧洲分子生物学实验室 用于基因治疗的修饰病毒粒子
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
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
CA3216491A1 (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
US20240207452A1 (en) 2021-04-23 2024-06-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
JP2024523707A (ja) 2021-07-08 2024-06-28 テナヤ セラピューティクス， インコーポレイテッド 遺伝子治療のための最適化された発現カセット
EP4405373A1 (en) * 2021-09-21 2024-07-31 California Institute of Technology Viral compositions for targeting the nervous system and the lung
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023073526A1 (en) 2021-10-25 2023-05-04 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
TW202334436A (zh) * 2021-11-02 2023-09-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
EP4442830A1 (en) 2021-11-29 2024-10-09 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
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
WO2023164545A1 (en) 2022-02-23 2023-08-31 Massachusetts Institute Of Technology Methods for upregulating shank3 expression
WO2023205397A1 (en) * 2022-04-22 2023-10-26 The Children's Hospital Of Philadelphia Human ependyma-specific promoter and uses thereof
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
TW202421787A (zh) 2022-09-06 2024-06-01 美商特納亞治療股份有限公司 保護心臟之心臟病療法
WO2024054983A1 (en) 2022-09-08 2024-03-14 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
WO2024175604A1 (en) 2023-02-21 2024-08-29 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
WO2024194491A1 (en) 2023-03-22 2024-09-26 Ospedale San Raffaele S.R.L. Gene therapy
WO2024199478A1 (en) * 2023-03-31 2024-10-03 Exegenesis Bio Co. Variant aav9 capsid proteins and uses 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

Family Cites Families (32)

* 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
US6228646B1 (en) 1996-03-07 2001-05-08 The Regents Of The University Of California 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
WO2001055361A2 (en) 2000-01-26 2001-08-02 Chiron Corporation Recombinant aav packaging systems
WO2001092551A2 (en) 2000-06-01 2001-12-06 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
WO2004083441A2 (en) 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts 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
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
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
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
WO2008103993A2 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
ES2615180T3 (es) 2007-07-14 2017-06-05 University Of Iowa Research Foundation Métodos y composiciones para el tratamiento de enfermedades cerebrales
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
CN103492574B (zh) 2011-02-22 2015-12-09 加州理工学院 使用腺相关病毒（aav）载体递送蛋白
RS62795B1 (sr) 2011-04-22 2022-02-28 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
WO2013029030A1 (en) 2011-08-24 2013-02-28 The Board Of Trustees Of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
CA2923352C (en) 2013-09-19 2022-05-03 Crucell Holland B.V. Stable adenovirus formulations
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
HUE054768T2 (hu) 2014-05-02 2021-09-28 Genzyme Corp AAV vektorok retina és CNS génterápiára
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2018204797A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav

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CN108699565B (zh) 2023-08-08
JP2019506141A (ja) 2019-03-07
CA3007495C (en) 2023-04-11
HK1257563A1 (zh) 2019-10-25
EP3387137B1 (en) 2021-02-03
EP3387137A4 (en) 2019-06-12
WO2017100671A1 (en) 2017-06-15
CO2018007203A2 (es) 2018-09-20
US20230295659A1 (en) 2023-09-21
MX2018006840A (es) 2019-03-28
EP3875593A1 (en) 2021-09-08
ZA201803808B (en) 2020-11-25
ES2869284T3 (es) 2021-10-25
EP3387137A1 (en) 2018-10-17
KR20180097629A (ko) 2018-08-31
CL2018001522A1 (es) 2018-12-21
US11499165B2 (en) 2022-11-15
RU2018120736A3 (pt) 2020-01-13
JP7066619B2 (ja) 2022-05-13
RU2727672C2 (ru) 2020-07-22
CA3007495A1 (en) 2017-06-15
IL259842A (en) 2018-07-31
RU2018120736A (ru) 2020-01-13
US20170166926A1 (en) 2017-06-15
IL259842B (en) 2022-08-01
CN108699565A (zh) 2018-10-23
SG11201804713VA (en) 2018-07-30

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