PL3254703T3 - Wiriony wirusa towarzyszącego adenowirusom z różnymi kapsydami i sposoby ich zastosowania - Google Patents

Wiriony wirusa towarzyszącego adenowirusom z różnymi kapsydami i sposoby ich zastosowania

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Publication number
PL3254703T3
PL3254703T3 PL17168534T PL17168534T PL3254703T3 PL 3254703 T3 PL3254703 T3 PL 3254703T3 PL 17168534 T PL17168534 T PL 17168534T PL 17168534 T PL17168534 T PL 17168534T PL 3254703 T3 PL3254703 T3 PL 3254703T3
Authority
PL
Poland
Prior art keywords
adeno
methods
associated virus
variant capsid
virus virions
Prior art date
Application number
PL17168534T
Other languages
English (en)
Inventor
David V. Schaffer
Ryan R. Klimczak
James T. Koerber
John G. Flannery
Deniz DALKARA MOUROT
Meike VISEL
Leah C.T. BYRNE
Original Assignee
The Regents Of The University Of California
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 The Regents Of The University Of California filed Critical The Regents Of The University Of California
Publication of PL3254703T3 publication Critical patent/PL3254703T3/pl

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Families Citing this family (318)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use 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
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
US10266845B2 (en) * 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
EP2996725A4 (en) * 2013-05-16 2016-12-28 Applied Genetic Tech Corp PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS AND METHODS FOR TREATING COLOR BLINDNESS AND OTHER PROBLEMS
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
WO2014207190A1 (en) * 2013-06-28 2014-12-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for expressing a polynucleotide of interest in the retina of a subject
EP3702461A1 (en) 2013-07-08 2020-09-02 Institut National De La Sante Et De La Recherche Medicale - Inserm Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
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
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015126972A1 (en) * 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US10307492B2 (en) * 2014-03-11 2019-06-04 Wayne State University Modified MGLUR6 promoter and methods of use
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
JP2017522895A (ja) * 2014-07-31 2017-08-17 ザ ボード オブ リージェンツ オブ ザ ユニヴァーシティ オブ オクラホマ 哺乳類ｒｐｅ６５の高イソメロヒドロラーゼ活性突然変異体
IL251178B (en) * 2014-09-16 2022-07-01 Genzyme Corp Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc)
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
EP3256170B1 (en) * 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
CA2977355A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
FI3265178T3 (fi) * 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
JP6836999B2 (ja) * 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US20180140626A1 (en) * 2015-05-14 2018-05-24 Joslin Diabetes Center, Inc. Retinol-binding protein 3 (rbp3) as a protective factor in non-diabetic retinal degeneration
EP3929287A3 (en) 2015-06-18 2022-04-13 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
JP6966442B2 (ja) * 2015-12-04 2021-11-17 ソルボンヌ・ユニヴェルシテSorbonne Universite プロモーター及びその使用
EP3387434A1 (en) 2015-12-11 2018-10-17 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin c type, and deficiency of mmachc
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
IL262207B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
ES2908467T3 (es) 2016-04-29 2022-04-29 Adverum Biotechnologies Inc Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante
EP3448411A1 (en) 2016-04-29 2019-03-06 Gensight Biologics SA Optogenetic visual restoration using chrimson
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017192750A1 (en) * 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CN117531025A (zh) * 2016-06-16 2024-02-09 阿德夫拉姆生物技术股份有限公司 使用aav2变体和阿柏西普治疗amd
PL3472317T3 (pl) * 2016-06-16 2022-08-08 Adverum Biotechnologies, Inc. Kompozycje i sposoby zmniejszania neowaskularyzacji oka
CN109640949B (zh) * 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
WO2018044912A1 (en) 2016-08-29 2018-03-08 Wayne State University Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
EP3535282A4 (en) * 2016-11-06 2020-04-08 Nanoscope Technologies LLC OPTOGENETIC MODULATION BY OPSINS WITH MULTIPLE CHARACTERISTICS FOR RESTORATION OF VISION AND OTHER ASSOCIATED APPLICATIONS
US11236360B2 (en) 2016-12-09 2022-02-01 Regents Of The University Of Minnesota Adeno-associated viruses engineered for selectable tropism
JP2020508289A (ja) 2017-01-31 2020-03-19 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
JP2020510424A (ja) * 2017-02-28 2020-04-09 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 改変ａａｖカプシドおよびその使用
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
EP4219806A3 (en) * 2017-04-11 2023-09-27 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library
KR20240093919A (ko) 2017-04-14 2024-06-24 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR20250023600A (ko) * 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
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.
AU2018350700B2 (en) * 2017-10-16 2025-04-24 VeonGen Therapeutics GmbH AAV vectors
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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
MX2020003945A (es) 2017-10-18 2020-11-09 Regenxbio Inc Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente .
KR20200067195A (ko) 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
KR102763312B1 (ko) * 2017-10-20 2025-02-07 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법
WO2019084015A1 (en) * 2017-10-23 2019-05-02 Daniel Schmidt PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
EP3749754A4 (en) * 2018-02-08 2021-10-20 Applied Stemcell, Inc. TARGET GENE VARIANT SCREENING METHODS
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
IL277234B2 (en) 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
MX2020010465A (es) * 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3098566A1 (en) 2018-04-29 2019-11-07 Zhuchun WU Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
IL278598B2 (en) * 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
IL319002A (en) 2018-06-14 2025-04-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
EP3808840A4 (en) * 2018-06-14 2022-03-23 Avixgen Inc. Pharmaceutical composition comprising fusion protein of cell-penetrating peptide and rpe65 for treatment of leber's congenital amaurosis
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
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
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
AU2019354296A1 (en) 2018-10-01 2021-04-29 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
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
JP2022514112A (ja) 2018-10-15 2022-02-09 リジェネクスバイオ インコーポレイテッド 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法
BR112021009913A2 (pt) * 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica
CA3122272A1 (en) 2018-12-12 2020-06-18 Tesseract Health, Inc. Optical apparatus and associated devices for biometric identification and health status determination
CN113825521A (zh) 2018-12-21 2021-12-21 美利坚合众国由健康及人类服务部部长代表 腺相关病毒及其用于内耳疗法的用途
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
KR20210126096A (ko) 2019-02-15 2021-10-19 상가모 테라퓨틱스, 인코포레이티드 재조합 aav를 제조하기 위한 조성물 및 방법
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CN111621502B (zh) * 2019-02-28 2023-05-02 武汉纽福斯生物科技有限公司 视网膜劈裂蛋白的编码序列、其表达载体构建及其应用
EP3930694A1 (en) 2019-02-28 2022-01-05 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
WO2020180951A1 (en) 2019-03-04 2020-09-10 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
EP3715358A1 (en) * 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP4667578A3 (en) 2019-04-03 2026-04-01 REGENXBIO Inc. Gene therapy for eye pathologies
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
ES2965341T3 (es) 2019-04-11 2024-04-12 Regenxbio Inc Métodos de cromatografía de exclusión por tamaño para la caracterización de composiciones de virus adenoasociados recombinantes
JP7630443B2 (ja) 2019-04-19 2025-02-17 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
US20220195462A1 (en) 2019-04-24 2022-06-23 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
CA3132447A1 (en) 2019-04-24 2020-10-29 Takara Bio Inc. Aav mutant having brain-targeting property
TWI862583B (zh) * 2019-04-26 2024-11-21 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
JP2022530845A (ja) * 2019-04-27 2022-07-04 オクジェン アイエヌシー． 眼疾患に対するａｄｅｎｏ随伴ウイルス連ウイルスベクター媒介遺伝子治療
US11737665B2 (en) 2019-06-21 2023-08-29 Tesseract Health, Inc. Multi-modal eye imaging with shared optical path
CN114269352A (zh) * 2019-06-27 2022-04-01 佛罗里达大学研究基金会 用透明质酸增强aav介导的眼组织转导
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
EP3997214B1 (en) * 2019-07-11 2023-08-02 Centre National de la Recherche Scientifique Chemically-modified adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
RU2751592C2 (ru) 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
CA3149401A1 (en) 2019-08-26 2021-03-04 Stephen Joseph Pakola Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab
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
FI4027983T3 (fi) 2019-09-11 2026-02-03 Adverum Biotechnologies Inc Silmän neovaskulaaristen sairauksien hoito afliberseptiä koodaavilla aav2-varianteilla
CN114375195A (zh) 2019-09-11 2022-04-19 阿德夫拉姆生物技术股份有限公司 使用编码阿柏西普的aav2变体治疗眼新血管疾病的方法
US20220325296A1 (en) * 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021071835A1 (en) 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
WO2021073568A1 (en) * 2019-10-16 2021-04-22 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
CN114729009A (zh) * 2019-10-31 2022-07-08 伯尔尼大学 用于眼部基因递送的aav载体变体
US12521451B2 (en) * 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
CN115335529B (zh) * 2019-11-26 2026-07-03 马萨诸塞大学 用于递送kh902(康柏西普)的重组腺相关病毒及其用途
AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
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
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
KR20220131522A (ko) 2020-01-22 2022-09-28 리젠엑스바이오 인크. 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료
BR112022015036A2 (pt) 2020-01-29 2022-10-11 Regenxbio Inc Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
US20230088992A1 (en) 2020-01-29 2023-03-23 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
AU2021218703A1 (en) 2020-02-14 2022-07-28 Ultragenyx Pharmaceutical Inc. Gene therapy for treating CDKL5 deficiency disorder
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
US12686874B2 (en) 2020-03-31 2026-07-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2021204407A1 (en) 2020-04-10 2021-10-14 Sorbonne Université G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4138929A1 (en) 2020-04-24 2023-03-01 Institut National de la Santé et de la Recherche Médicale (INSERM) Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy
CA3175905A1 (en) 2020-04-27 2021-11-04 David H. Kirn Codon optimized gla genes and uses thereof
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2021243085A2 (en) 2020-05-29 2021-12-02 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions
EP4199972A4 (en) * 2020-08-21 2025-01-15 Capsida, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS WITH PREFERRED EXPRESSION LEVELS
CN112226461B (zh) * 2020-08-21 2022-04-22 华侨大学 Cd4阳性细胞特异性基因传递载体及其应用
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
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
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
EP4225777A2 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Adeno-associated viruses for ocular delivery of gene therapy
TW202228648A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如高黏度調配物之用於脈絡膜上投與之調配物
TW202228646A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如凝膠調配物之用於脈絡膜上投與之調配物
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
KR20230098176A (ko) 2020-10-29 2023-07-03 리젠엑스바이오 인크. 안구 적응증을 위한 벡터화된 tnf-알파 길항제
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
AU2021378270A1 (en) * 2020-11-10 2023-06-15 Avirmax Biopharma Inc. Engineered viral capsids and methods of use
US20240002839A1 (en) 2020-12-02 2024-01-04 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
EP4263841A1 (en) 2020-12-16 2023-10-25 REGENXBIO Inc. Method of producing a recombinant adeno-associated virus particle
US20240067678A1 (en) * 2020-12-16 2024-02-29 Children's Medical Research Institute Adeno-associated virus capsids and vectors
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
AU2022210592A1 (en) 2021-01-21 2023-07-13 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
AU2022221284A1 (en) 2021-02-10 2023-08-17 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
CN113121651B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 低中和抗体腺相关病毒衣壳蛋白
CN113121652B (zh) * 2021-04-19 2022-10-11 上海信致医药科技有限公司 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用
ES3034370T3 (en) 2021-04-20 2025-08-18 Inst Nat Sante Rech Med Compositions and methods for treating retinal degenerative disorders
IL307902A (en) 2021-04-26 2023-12-01 Regenxbio Inc Microdystrophin gene therapy administration for treatment of dystrophinopathies
CN117222400A (zh) 2021-04-27 2023-12-12 阿德夫拉姆生物技术股份有限公司 使用编码阿柏西普的aav2变体治疗眼部疾病的方法
EP4334454A2 (en) 2021-05-04 2024-03-13 RegenxBio Inc. Novel aav vectors and methods and uses thereof
US20240358857A1 (en) 2021-05-11 2024-10-31 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
WO2022248634A1 (en) 2021-05-26 2022-12-01 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
EP4349852A4 (en) * 2021-05-28 2025-09-03 Shanghai Regenelead Therapies Co Ltd RECOMBINANT ADENO-ASSOCIATED VIRUS HAVING A VARIANT CAPSID, AND ITS APPLICATION
AU2022283898A1 (en) * 2021-06-03 2024-01-04 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
EP4366757A1 (en) 2021-07-07 2024-05-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
CR20240004A (es) 2021-07-08 2024-06-11 Biocad Joint Stock Co Composición farmacéutica de virus no encapsulado
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
AU2022313258A1 (en) 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease
JP2024531138A (ja) 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
AR126840A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
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
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
US20230338581A1 (en) 2021-10-11 2023-10-26 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN120187407A (zh) * 2021-12-15 2025-06-20 健达九州(北京)生物科技有限公司 重组aav载体及其用途
JP2025506383A (ja) 2022-02-02 2025-03-11 アドヴェラム バイオテクノロジーズ， インコーポレイテッド アフリベルセプトをコードするａａｖ２バリアントを使用して眼内血管新生疾患を処置する方法
KR20240161978A (ko) 2022-02-16 2024-11-13 애드베룸 바이오테크놀로지스, 인코포레이티드 재조합 아데노 관련 바이러스에 의한 신혈관성 amd의 cst 변동을 감소시키는 방법
KR20240152351A (ko) 2022-02-17 2024-10-21 스카이라인 테라퓨틱스 리미티드 변형된 aav 캡시드 폴리펩티드를 갖는 재조합 아데노-연관 바이러스
CA3245327A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. MODIFIED BATCH-BASED AAV PRODUCTION SYSTEMS AND PROCESSES
EP4493226A1 (en) 2022-03-13 2025-01-22 RegenxBio Inc. Modified muscle-specific promoters
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
CA3255242A1 (en) * 2022-03-30 2023-10-05 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated viral vectors for the delivery of nucleic acids across retinal regions
TW202346600A (zh) * 2022-03-30 2023-12-01 高等教育聯邦系統 匹茲堡大學 用於將核酸遞送至視網膜神經節細胞和/或視網膜色素上皮細胞的腺相關病毒載體
US20250327092A1 (en) * 2022-03-30 2025-10-23 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells
EP4499669A4 (en) * 2022-03-30 2026-06-24 Univ Pittsburgh Commonwealth Sys Higher Education Adenoid-associated viral vectors
AU2023250660A1 (en) 2022-04-06 2024-10-24 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN120590489A (zh) * 2022-04-12 2025-09-05 合肥星眸生物科技有限公司 一种融合型腺相关病毒及其应用
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
CN116970648A (zh) * 2022-04-24 2023-10-31 上海朗昇生物科技有限公司 新型aav衣壳改造株及其用途
TW202417633A (zh) 2022-05-03 2024-05-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗TNF-α抑制劑
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
CA3252767A1 (en) 2022-06-07 2023-12-14 Adverum Biotechnologies, Inc. VARIANTS OF MELANOPSINE FOR VISION RESTORATION
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
JP2025528188A (ja) 2022-08-11 2025-08-26 インスティテュート オブ モレキュラー アンド クリニカル オフサルモロジー バーゼル （アイオービー） ヒト錐体光受容体オプトジェネティクス構築物
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
KR20250115381A (ko) 2022-10-20 2025-07-30 스페어링비젼 망막 변성 질환 치료를 위한 조성물 및 방법
EP4357359A1 (en) 2022-10-20 2024-04-24 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
JP2025538387A (ja) 2022-11-16 2025-11-28 リジェネロン・ファーマシューティカルズ・インコーポレイテッド プロテアーゼ切断可能なリンカーを有する膜結合ｉｌ－１２を含むキメラタンパク質
WO2024146935A1 (en) 2023-01-06 2024-07-11 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
CN118359687A (zh) * 2023-01-18 2024-07-19 上海朗昇生物科技有限公司 一种新衣壳蛋白变体以及其用途
KR20250142340A (ko) 2023-02-02 2025-09-30 퀘세라 리미티드 재조합 아데노-부속 바이러스 벡터
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024211831A1 (en) 2023-04-05 2024-10-10 Adverum Biotechnologies, Inc. Immune landscape signatures for ocular inflammation
JP2026513932A (ja) 2023-04-07 2026-05-01 リジェネクスバイオ インコーポレイテッド 組換えａａｖ生成のための組成物及び方法
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
EP4694904A2 (en) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024229389A1 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
JP2026515970A (ja) * 2023-05-04 2026-05-19 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カプシドバリアント及びその使用
EP4709866A2 (en) 2023-05-07 2026-03-18 REGENXBIO Inc. Compositions and methods for recombinant aav production
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
CN121219325A (zh) * 2023-05-23 2025-12-26 北京因诺惟康医药科技有限公司 一种腺相关病毒衣壳蛋白、包含其的腺相关病毒、载体及应用
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
KR20260039752A (ko) 2023-07-13 2026-03-20 크리야 테라퓨틱스, 인크. 보체 경로 억제제 융합체 및 그의 사용 방법
CN117230117A (zh) * 2023-08-04 2023-12-15 中国科学院深圳先进技术研究院 重组腺相关病毒包装质粒、突变体及其制备方法和应用
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
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
CN117143203B (zh) * 2023-09-06 2026-03-03 广州译码基因科技有限公司 可提高aav病毒全眼感染能力的衣壳蛋白突变体及其应用
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
CN117247434B (zh) * 2023-11-10 2024-02-02 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
AU2024386366A1 (en) 2023-11-23 2026-06-04 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025129085A1 (en) 2023-12-15 2025-06-19 Port Therapeutics, Inc. Use of thermal bioswitches in aav-based gene therapy
CN119080889B (zh) * 2024-09-18 2025-06-24 广州译码基因科技有限公司 提高aav对眼组织感染能力的衣壳蛋白突变体及应用
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
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025217230A1 (en) * 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025214408A1 (en) 2024-04-10 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Optogenetic modulation for vision restoration
WO2025214404A1 (en) 2024-04-10 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Optogenetic modulation for vision restoration
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
CN121046460A (zh) * 2024-05-31 2025-12-02 上海朗昇生物科技有限公司 用于治疗Prom1相关视网膜疾病的新型治疗药物
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
TW202607144A (zh) 2024-07-16 2026-02-16 美商愛德維仁生物科技公司 使用編碼阿柏西普（ａｆｌｉｂｅｒｃｅｐｔ）之ａａｖ２變異體治療眼部新生血管性疾病的方法
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026080515A2 (en) 2024-10-08 2026-04-16 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cells and cell lines and methods of use thereof
CN118955654B (zh) * 2024-10-15 2025-05-16 朗信启昇(苏州)生物制药有限公司 具有变异衣壳的腺相关病毒病毒体及其用途
WO2026102076A1 (en) 2024-11-07 2026-05-15 Juno Therapeutics, Inc. Aav production method
WO2026114267A1 (en) 2024-11-29 2026-06-04 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating alzheimer's disease in primates
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
EP4763209A1 (en) 2024-12-17 2026-06-24 Institut National de la Santé et de la Recherche Médicale Use of (alpha)s-setmar agonist for preventing or treating glioblastoma
WO2026137014A1 (en) 2024-12-20 2026-06-25 Samsung Bioepis Co., Ltd. Methods of purifying recombinant adeno-associated viruses
CN122255230A (zh) * 2026-05-12 2026-06-23 沈阳兴齐眼药股份有限公司 Aav衣壳蛋白变体及其应用

Family Cites Families (103)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
US5639872A (en) 1993-07-27 1997-06-17 Hybridon, Inc. Human VEGF-specific oligonucleotides
US6440425B1 (en) 1995-05-01 2002-08-27 Aventis Pasteur Limited High molecular weight major outer membrane protein of moraxella
US6001650A (en) * 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
WO1997020925A1 (en) 1995-12-08 1997-06-12 Hybridon, Inc. Modified vegf antisense oligonucleotides for treatment of skin disorders
US6096548A (en) 1996-03-25 2000-08-01 Maxygen, Inc. Method for directing evolution of a virus
WO1997038723A1 (en) 1996-04-16 1997-10-23 Immusol Incorporated Targeted viral vectors
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
US6551795B1 (en) * 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
CN1159445C (zh) * 1998-09-11 2004-07-28 加利福尼亚大学董事会 能在心脏中组织特异性表达的重组腺病毒
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
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
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US8232081B2 (en) 1999-09-21 2012-07-31 Basf Se Methods and microorganisms for production of panto-compounds
DE10001363A1 (de) 2000-01-14 2001-07-26 Windmoeller & Hoelscher Extruderdüsenkopf
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
WO2002024897A2 (en) * 2000-09-22 2002-03-28 Virxsys Conditionally replicating viral vectors and their use
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
US20030129203A1 (en) * 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
US20050106558A1 (en) 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
DE10216005A1 (de) 2002-04-11 2003-10-30 Max Planck Gesellschaft Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
US7254489B2 (en) 2002-05-31 2007-08-07 Microsoft Corporation Systems, methods and apparatus for reconstructing phylogentic trees
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
WO2004056080A1 (en) * 2002-12-18 2004-07-01 Novartis Ag Endothelial cell specifically binding peptides
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
CA2524499A1 (en) * 2003-05-14 2004-11-25 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti Spa Broadening adenovirus tropism
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US7368428B2 (en) 2003-06-23 2008-05-06 A&G Pharmaceutical, Inc. Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis
US9441244B2 (en) * 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use 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
AU2004276316A1 (en) 2003-09-24 2005-04-07 The Board Of Trustees Of The Leland Stanford Junior University IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
KR100550967B1 (ko) 2003-12-03 2006-02-13 주식회사 엘지생활건강 유중수형 화장료 조성물
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
US20060211650A1 (en) 2004-12-16 2006-09-21 Forest Laboratories, Inc. Reducing carbohydrate derivatives of adamantane amines, and synthesis and methods of use thereof
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP2009502140A (ja) 2005-07-22 2009-01-29 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 光活性化陽イオンチャネルおよびその使用
CN1834255A (zh) * 2005-12-23 2006-09-20 上海交通大学附属第一人民医院 加快并提高腺相关病毒介导的基因在视网膜细胞内表达的方法
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8470790B2 (en) * 2006-05-04 2013-06-25 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
WO2007148971A2 (en) * 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
CN1966082B (zh) * 2006-11-03 2010-06-30 许瑞安 一种防治结直肠癌的基因药物及其制备方法和用途
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
RU2571931C2 (ru) 2007-04-13 2015-12-27 Каталист Биосайенсис, Инк. Полипептиды на основе модифицированного фактора vii и их применение
EP1985708B1 (en) * 2007-04-27 2015-04-15 Universität Rostock Selective targeting of viruses to neural precursor cells
US8632764B2 (en) * 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
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
CN101532024A (zh) * 2009-04-30 2009-09-16 许瑞安 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
US8663624B2 (en) * 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA2826273C (en) * 2011-02-10 2021-11-02 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
DK3693025T3 (da) * 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
US20180043034A1 (en) 2015-03-06 2018-02-15 Massachusetts Eye And Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
KR20250023600A (ko) * 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.

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