ZA201803808B - Targeting peptides for directing adeno-associated viruses (aavs) - Google Patents

Targeting peptides for directing adeno-associated viruses (aavs)

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Publication number
ZA201803808B
ZA201803808B ZA201803808A ZA201803808A ZA201803808B ZA 201803808 B ZA201803808 B ZA 201803808B ZA 201803808 A ZA201803808 A ZA 201803808A ZA 201803808 A ZA201803808 A ZA 201803808A ZA 201803808 B ZA201803808 B ZA 201803808B
Authority
ZA
South Africa
Prior art keywords
aavs
adeno
directing
associated viruses
targeting peptides
Prior art date
Application number
ZA201803808A
Other languages
English (en)
Inventor
Benjamin E Deverman
Viviana Gradinaru
Ken Y Chan
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 ZA201803808B publication Critical patent/ZA201803808B/en

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    • 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
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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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • 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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ZA201803808A 2015-12-11 2018-06-07 Targeting peptides for directing adeno-associated viruses (aavs) ZA201803808B (en)

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
PCT/US2016/065969 WO2017100671A1 (en) 2015-12-11 2016-12-09 TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)

Publications (1)

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ZA201803808B true ZA201803808B (en) 2020-11-25

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ZA201803808A ZA201803808B (en) 2015-12-11 2018-06-07 Targeting peptides for directing adeno-associated viruses (aavs)

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Families Citing this family (155)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JP6754361B2 (ja) * 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
AU2016366549B2 (en) 2015-12-11 2022-11-10 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
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
FI3574091T3 (fi) 2017-01-30 2023-05-03 Brainvectis Ekspressiovektori kolesterolin 24-hydrolaasia varten polyglutamiinin toiston spinoserebellaariataksioiden hoidossa
MX2019012113A (es) 2017-04-14 2020-09-10 Univ Nat Taiwan Terapia génica para la deficiencia de la aadc.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
TWI722310B (zh) * 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
US20190085358A1 (en) * 2017-08-25 2019-03-21 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
US20200263199A1 (en) * 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR102776626B1 (ko) * 2017-10-16 2025-03-04 비게네론 게엠베하 Aav 벡터
US11458129B2 (en) 2017-11-02 2022-10-04 California Institute Of Technology Neurokinin antagonists and uses thereof
EP3703716A4 (en) 2017-11-02 2021-12-01 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
JP7430652B2 (ja) 2018-03-05 2024-02-13 ザ チルドレンズ メディカル センター コーポレーション 蝸牛および前庭細胞に核酸を送達するための組成物および方法
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
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
US12121563B2 (en) 2018-04-09 2024-10-22 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
WO2019198084A1 (en) * 2018-04-12 2019-10-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Anti-aging compositions and methods of use
WO2019210325A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
US20210230632A1 (en) * 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
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
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
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
CA3114621A1 (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
US20210348194A1 (en) 2018-10-05 2021-11-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
CA3115652A1 (en) 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス／Ｓｆ９システムにおけるｒＡＡＶの大規模産生のための発現ベクター
HUE060949T2 (hu) 2018-10-29 2023-04-28 Inst Nat Sante Rech Med Koleszterin 24-hidroláz expressziós vektor amiotrófiás laterálszklerózis kezelésében
US20210393713A1 (en) * 2018-11-20 2021-12-23 The Trustees Of The University Of Pennsylvania Compositions and methods useful for targeting the blood-brain barrier
WO2020150556A1 (en) 2019-01-18 2020-07-23 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
EP3917945A4 (en) * 2019-01-30 2023-02-08 The Broad Institute Inc. ADVANCED ADENO-ASSOCIATED VIRUS (AAVS) TARGETED DELIVERY SYSTEMS
JP2022523719A (ja) * 2019-01-31 2022-04-26 オレゴン・ヘルス・アンド・サイエンス・ユニバーシティ Ａａｖキャプシドの転写依存性定方向進化を使用するための方法
EP3923995A4 (en) * 2019-02-15 2023-05-17 Allen Institute ARTIFICIAL EXPRESSION RECOMBINANTS FOR THE SELECTIVE MODULATION OF GENE EXPRESSION IN SELECTED NEURON CELL POPULATIONS
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
CN113874387B (zh) * 2019-03-28 2025-01-07 总医院公司 用于转基因表达的工程化腺相关病毒(aav)载体
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
WO2020223274A1 (en) 2019-04-29 2020-11-05 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
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3972631B1 (en) * 2019-05-21 2024-07-10 Institut National de la Santé et de la Recherche Médicale (INSERM) 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
US20220290182A1 (en) 2019-08-09 2022-09-15 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
CA3151087A1 (en) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
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
US20230392163A1 (en) * 2019-10-17 2023-12-07 Ginkgo Bioworks Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US20210162002A1 (en) * 2019-10-17 2021-06-03 The Penn State Research Foundation Regenerating functional neurons for treatment of spinal cord injury and als
IL292264B2 (en) * 2019-10-17 2025-10-01 Stridebio Inc aav transfer cassette
EP4061946A4 (en) * 2019-11-19 2024-03-06 Asklepios Biopharmaceutical, Inc. THERAPEUTIC ADENO-ASSOCIATED VIRUS INCLUDING LIVER-SPECIFIC PROMOTERS TO TREAT PUMP DISEASE AND LYSOSOMAL DISORDERS
CN115023242B (zh) * 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
WO2021142300A2 (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
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
IL295529A (en) 2020-02-13 2022-10-01 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
BR112022021134A2 (pt) * 2020-04-20 2023-02-14 Tenaya Therapeutics Inc Vírus adeno-associado com capsídeo projetado
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
CA3181011A1 (en) * 2020-04-28 2021-11-04 Genethon Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders
EP4150626A1 (en) 2020-04-29 2023-03-22 The Broad Institute Inc. Machine learning accelerated protein engineering through fitness prediction
CN115867646A (zh) * 2020-05-01 2023-03-28 博德研究所 工程化中枢神经系统组合物
KR20230022175A (ko) 2020-05-13 2023-02-14 보이저 테라퓨틱스, 인크. Aav 캡시드의 향성 방향변경
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
US20230227802A1 (en) 2020-07-27 2023-07-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
GB202011871D0 (en) * 2020-07-30 2020-09-16 Cambridge Entpr Ltd Composition and method
US20230257431A1 (en) 2020-08-05 2023-08-17 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
EP4192487A4 (en) 2020-08-07 2024-10-02 Spacecraft Seven, LLC GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR
MX2023001998A (es) 2020-08-17 2023-05-04 Massachusetts Inst Technology Enfoques de terapia con el gen shank3.
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
CA3192600A1 (en) * 2020-08-21 2022-02-24 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
WO2022061378A2 (en) * 2020-09-21 2022-03-24 Asklepios Biopharmaceutical, Inc. Methods for treating neurological disease
CN112194706B (zh) * 2020-09-30 2022-03-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
AR123776A1 (es) 2020-10-15 2023-01-11 Hoffmann La Roche Construcciones de ácido nucleico para la activación simultánea de genes
WO2022101363A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
EP4256065A2 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
EP4301768A2 (en) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2024511808A (ja) * 2021-03-29 2024-03-15 南京大学 ウイルスベクターに基づくｒｎａ送達システム及びその使用
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
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
US20240382617A1 (en) * 2021-09-21 2024-11-21 Capsida Biotherapeutics, Inc. Virus compositions to efficiently target the nervous system and the lungs
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4422613A1 (en) 2021-10-25 2024-09-04 Novartis AG Methods for improving adeno-associated virus (aav) delivery
US20250001012A1 (en) 2021-11-02 2025-01-02 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
EP4469585A2 (en) 2022-01-25 2024-12-04 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
TW202342526A (zh) 2022-02-21 2023-11-01 大陸商上海瑞宏迪醫藥有限公司 Ｖｅｇｆ結合分子及其醫藥用途
WO2023164545A1 (en) 2022-02-23 2023-08-31 Massachusetts Institute Of Technology Methods for upregulating shank3 expression
IL316430A (en) * 2022-04-22 2024-12-01 Childrens Hospital Philadelphia HUMAN EPENDYMA-SPECIFIC PROMOTER AND ITS USES
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
EP4615492A1 (en) 2022-11-08 2025-09-17 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
CN120936709A (zh) * 2023-03-09 2025-11-11 新加坡科技研究局 经工程化以增强组织转导和特异性的腺相关病毒的新组合物
AU2024234833A1 (en) 2023-03-10 2025-10-23 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
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024229389A1 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229425A1 (en) * 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
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
WO2025106874A1 (en) * 2023-11-15 2025-05-22 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
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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

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
JP2001500014A (ja) 1996-09-06 2001-01-09 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 組換えアデノ随伴ウイルスの生産のためにｃｒｅ―ｌｏｘを用いる方法
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
EP1257656A2 (en) 2000-01-26 2002-11-20 Chiron Corporation Recombinant aav packaging systems
ES2505700T3 (es) 2000-06-01 2014-10-10 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados
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
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
EP3910063A1 (en) 2003-09-30 2021-11-17 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
RU2457252C2 (ru) * 2006-06-21 2012-07-27 Амстердам Молекьюла Терапьютикс (Амт) Ип Б.В. AAV ВЕКТОРЫ С УСОВЕРШЕНСТВОВАННЫМИ Rep-КОДИРУЮЩИМИ ПОСЛЕДОВАТЕЛЬНОСТЯМИ, ИСПОЛЬЗУЕМЫМИ В СИСТЕМАХ ПРОДУКЦИИ НА ОСНОВЕ КЛЕТОК НАСЕКОМЫХ
WO2008103993A2 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
EP2176283B1 (en) * 2007-07-14 2016-11-02 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
US20110223635A1 (en) 2008-08-11 2011-09-15 Karl Deisseroth Method and Composition for Controlling Gene Expression
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system 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
ES2638342T3 (es) 2011-04-22 2017-10-19 The Regents Of The University Of California Viriones de virus adenoasociado con cápside variante y métodos para su uso
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
ES2711115T3 (es) 2013-09-19 2019-04-30 Janssen Vaccines & Prevention Bv Formulaciones de adenovirus mejoradas
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
PE20170260A1 (es) * 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
CN119491003A (zh) * 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CN110914427B (zh) * 2017-05-05 2024-08-09 沃雅戈治疗公司 调节性多核苷酸
EP3618839A4 (en) * 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
TWI722310B (zh) * 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法

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