KR102202908B1 - 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법 - Google Patents

변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법 Download PDF

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KR102202908B1
KR102202908B1 KR1020207002952A KR20207002952A KR102202908B1 KR 102202908 B1 KR102202908 B1 KR 102202908B1 KR 1020207002952 A KR1020207002952 A KR 1020207002952A KR 20207002952 A KR20207002952 A KR 20207002952A KR 102202908 B1 KR102202908 B1 KR 102202908B1
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gly
ser
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ala
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KR20200016988A (ko
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데이빗 브이. 스캐퍼
라얀 알. 클림잭
제임스 티. 코에르버
존 지. 플랜너리
무롯 데니즈 달카라
마이크 비젤
레아 씨.티. 번
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더 리젠츠 오브 더 유니버시티 오브 캘리포니아
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Families Citing this family (293)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
LT3254703T (lt) * 2011-04-22 2020-05-25 The Regents Of The University Of California Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai
TWI775096B (zh) 2012-05-15 2022-08-21 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)
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
US10266845B2 (en) * 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
DK2954051T3 (da) 2013-02-08 2019-07-08 Univ Pennsylvania Modificeret kapsid til genoverførsel til behandling af nethinden
JP6591956B2 (ja) 2013-03-15 2019-10-16 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 Ｍｐｓ１を治療するための組成物および方法
CA2912525A1 (en) * 2013-05-16 2014-11-20 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
WO2014194132A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
DK3011031T3 (da) 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
JP6625971B2 (ja) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化
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
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
ES2716615T3 (es) 2013-06-28 2019-06-13 Inst Nat Sante Rech Med Métodos para expresar un polinucleótido de interés en la retina de un sujeto
JP6571075B2 (ja) 2013-07-08 2019-09-04 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル アンチセンスオリゴヌクレオチド介在性エクソンスキッピングを、それを必要とする対象の網膜において行うための方法
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
IL289736B2 (en) * 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
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
WO2015089351A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Ｈｂｖ 및 바이러스 질병 및 질환을 위한 ｃｒｉｓｐｒ­ｃａｓ 시스템 및 조성물의 전달，용도 및 치료적 적용
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US20170007720A1 (en) * 2014-02-21 2017-01-12 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
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
CA3234028A1 (en) * 2014-03-11 2015-09-17 Wayne State University A modified mglur6 promoter and methods of use
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3126495A1 (en) 2014-04-02 2017-02-08 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
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
EP3188762A4 (en) * 2014-07-31 2018-01-10 The Board of Regents of the University of Oklahoma High isomerohydrolase activity mutants of mammalian rpe65
EP3194601B1 (en) * 2014-09-16 2021-10-27 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 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)
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016131009A1 (en) * 2015-02-13 2016-08-18 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
ES2920850T3 (es) * 2015-02-20 2022-08-10 Univ Iowa Res Found Procedimientos y composiciones para el tratamiento de enfermedades oculares genéticas
KR20170137730A (ko) * 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
WO2016154344A1 (en) * 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
WO2016183585A1 (en) * 2015-05-14 2016-11-17 Joslin Diabetes Center, Inc. Retinol-binding protein 3 (rbp3) as a protective factor in non-diabetic retinal degeneration
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
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
KR102312253B1 (ko) * 2015-12-04 2021-10-15 쏘흐본느 유니베흐시테 프로모터 및 이의 용도
WO2017099838A1 (en) 2015-12-11 2017-06-15 The United States Of America, 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
WO2017100671A1 (en) * 2015-12-11 2017-06-15 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
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
AU2017257169B2 (en) 2016-04-29 2021-07-08 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017187272A1 (en) 2016-04-29 2017-11-02 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
MX2018013463A (es) 2016-05-13 2019-07-04 4D Molecular Therapeutics Inc Capsides variantes de virus adenoasociados y metodos de uso de las mismas.
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
DK3472317T3 (da) * 2016-06-16 2022-05-23 Adverum Biotechnologies Inc Sammensætninger og fremgangsmåder til reduktion af okulær vaskularisation
PT3795181T (pt) 2016-06-16 2025-11-05 Adverum Biotechnologies Inc Tratamento de dmi com utilização da variante aav2 com aflibercept
KR20230039779A (ko) * 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR102877327B1 (ko) 2016-08-29 2025-10-27 웨인 스테이트 유니버시티 개선된 광 민감성을 갖는 채널옵신 변이체에서의 돌연변이의 확인 및 이의 용도
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
US11180537B2 (en) * 2016-11-06 2021-11-23 Nancoscope Technologies LLC Optogenetic modulation by Multi-Characteristic Opsins for vision restoration and other applications
US11236360B2 (en) 2016-12-09 2022-02-01 Regents Of The University Of Minnesota Adeno-associated viruses engineered for selectable tropism
AU2018227483A1 (en) * 2017-02-28 2019-09-12 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
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
WO2018189244A1 (en) * 2017-04-11 2018-10-18 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library
US11613739B2 (en) 2017-04-14 2023-03-28 Regenxbio Inc. Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating 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
CN110461368A (zh) 2017-06-30 2019-11-15 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及其使用方法
AU2018298133A1 (en) 2017-07-06 2020-01-23 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
EP3676373A4 (en) 2017-08-28 2021-06-09 The Regents of The University of California Adeno-associated virus capsid variants and methods of use thereof
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
AU2018350700B2 (en) * 2017-10-16 2025-04-24 Vigeneron Gmbh AAV vectors
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079496A2 (en) 2017-10-18 2019-04-25 Regenxbio, Inc. FULLY HUMAN ANTIBODY-BASED THERAPEUTIC AGENTS HAVING POST-TRANSLATION MODIFICATION
EP3697449A1 (en) 2017-10-18 2020-08-26 REGENXBIO Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
US11723988B2 (en) 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
US11851671B2 (en) 2017-10-23 2023-12-26 Regents Of The University Of Minnesota Programmable assembly of virus composites for receptor-targeted gene delivery
JP7184894B2 (ja) 2017-11-27 2022-12-06 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019157395A1 (en) * 2018-02-08 2019-08-15 Applied Stemcells, Inc Methods for screening variant of target gene
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
CA3093702A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
WO2019212922A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
US12365865B2 (en) 2018-04-29 2025-07-22 Regenxbio Inc. Scalable clarification process for recombinant AAV production
BR112020022858A2 (pt) * 2018-05-11 2021-02-23 Massachusetts Eye And Ear Infirmary tropismo específico hepático de vírus adeno-associado
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病を治療するための組成物および方法
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
US12070702B2 (en) 2018-06-14 2024-08-27 Regenxbio Inc. Anion exchange chromatography for recombinant AAV production
WO2019240431A1 (ko) * 2018-06-14 2019-12-19 (주) 에빅스젠 세포 투과 펩티드 및 rpe65의 융합 단백질을 포함하는 레버 선천성 흑암시 치료용 약학적 조성물
CN112703198B (zh) 2018-07-11 2025-05-30 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
AU2019319976B2 (en) 2018-08-10 2025-08-14 Regenxbio Inc. Scalable method for recombinant AAV production
EP3856913A4 (en) 2018-09-26 2022-10-26 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
AR116569A1 (es) 2018-10-01 2021-05-19 Ultragenyx Pharmaceutical Inc Terapia génica para tratar la acidemia propiónica
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2020081415A1 (en) 2018-10-15 2020-04-23 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
CA3121177A1 (en) * 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
EP3893718A4 (en) 2018-12-12 2022-11-09 Tesseract Health, Inc. BIOMETRIC IDENTIFICATION SYSTEMS AND RELATED DEVICES
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
CN113728108A (zh) 2019-02-15 2021-11-30 桑格摩生物治疗股份有限公司 用于生产重组aav的组合物和方法
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法
EP3931334A1 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
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
JP7597380B2 (ja) 2019-03-04 2024-12-10 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 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与
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
CA3135609A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4667578A2 (en) 2019-04-03 2025-12-24 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
HUE064411T2 (hu) 2019-04-11 2024-03-28 Regenxbio Inc Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére
CN113993552A (zh) 2019-04-19 2022-01-28 再生生物股份有限公司 腺相关病毒载体制剂和方法
CA3132447A1 (en) 2019-04-24 2020-10-29 Takara Bio Inc. Aav mutant having brain-targeting property
SG11202111414RA (en) 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
TW202532428A (zh) * 2019-04-26 2025-08-16 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
WO2020222858A1 (en) * 2019-04-27 2020-11-05 Ocugen, Inc. Adeno-associated virus vector mediated gene therapy for ophthalmic diseases
US11737665B2 (en) 2019-06-21 2023-08-29 Tesseract Health, Inc. Multi-modal eye imaging with shared optical path
AU2020308909A1 (en) * 2019-06-27 2022-01-27 University Of Florida Research Foundation, Incorporated Enhancing AAV-mediated transduction of ocular tissues with hyaluronic acid
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
AU2020311618B2 (en) * 2019-07-11 2025-04-10 Centre Hospitalier Universitaire De Nantes 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
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
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
WO2021021661A1 (en) 2019-07-26 2021-02-04 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
MX2022002366A (es) 2019-08-26 2022-07-19 Regenxbio Inc Tratamiento de la retinopatía diabética con fab anti-vegf completamente humano modificado postraduccionalmente.
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
CN114729384A (zh) * 2019-09-12 2022-07-08 博德研究所 工程化腺相关病毒衣壳
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
KR20220081365A (ko) 2019-10-07 2022-06-15 리젠엑스바이오 인크. 아데노-연관 바이러스 벡터 약제학적 조성물 및 방법
WO2021073568A1 (en) * 2019-10-16 2021-04-22 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
WO2021084133A1 (en) * 2019-10-31 2021-05-06 Universität Bern Aav vector variants for ocular gene delivery
IL292605B2 (en) * 2019-11-08 2025-09-01 Regeneron Pharma CRISPR and AAV strategies for treating childhood X-linked retinoschisis
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
CN115335529A (zh) * 2019-11-26 2022-11-11 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
JP2023503637A (ja) 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
CN115279400A (zh) 2020-01-10 2022-11-01 布里格姆妇女医院 用于跨血脑屏障递送免疫治疗剂以治疗脑癌的方法和组合物
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
WO2021150570A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
AU2021214174A1 (en) 2020-01-29 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
JP2023512043A (ja) 2020-01-29 2023-03-23 レジェンクスバイオ インコーポレーテッド ムコ多糖症ｉｖａの治療
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
WO2021188892A1 (en) 2020-03-19 2021-09-23 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US20230159609A1 (en) * 2020-04-10 2023-05-25 Sorbonne Université G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
JP2023523211A (ja) 2020-04-24 2023-06-02 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 眼科遺伝子治療後の、導入遺伝子産物を発現する形質転換細胞に対する免疫応答の誘導を防止するための方法
CA3175905A1 (en) 2020-04-27 2021-11-04 David H. Kirn Codon optimized gla genes and uses thereof
PE20230767A1 (es) 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides de aav
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
JP2023528801A (ja) 2020-05-29 2023-07-06 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 バリアントキャプシドを有するアデノ随伴ウイルスビリオン及びその使用方法
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
CN117321213A (zh) * 2020-08-21 2023-12-29 卡西达公司 具有优选表达水平的腺相关病毒组合物
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
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
CN113754728B (zh) * 2020-09-30 2022-07-12 广州派真生物技术有限公司 腺相关病毒突变体及其应用
JP2023545424A (ja) 2020-10-07 2023-10-30 リジェネックスバイオ インコーポレイテッド ゲル製剤などの脈絡膜上投与用製剤
JP2023545722A (ja) 2020-10-07 2023-10-31 リジェネックスバイオ インコーポレイテッド 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス
MX2023004035A (es) 2020-10-07 2023-07-05 Regenxbio Inc Formulaciones para la administración supracoroidea tales como formulaciones de viscosidad alta.
CA3198368A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
CA3196439A1 (en) * 2020-11-10 2022-05-19 Avirmax, Inc. Engineered viral capsids and methods of use
WO2022120022A1 (en) 2020-12-02 2022-06-09 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cell lines and methods of use thereof
IL303614A (en) 2020-12-16 2023-08-01 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
TW202241943A (zh) 2020-12-29 2022-11-01 美商銳進科斯生物股份有限公司 Tau特異性抗體基因療法組合物、方法及其用途
EP4281568A1 (en) 2021-01-21 2023-11-29 RegenxBio Inc. Improved production of recombinant polypeptides and viruses
WO2022173605A2 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
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
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
WO2022223644A2 (en) 2021-04-20 2022-10-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for treating retinal degenerative disorders
TW202309293A (zh) 2021-04-26 2023-03-01 美商銳進科斯生物股份有限公司 用於治療肌縮蛋白症(dystrophinopathy)之微小肌縮蛋白基因療法給藥
US20240197915A1 (en) 2021-04-27 2024-06-20 Adverum Biotechnologies, Inc. Methods of treating ocular diseases using aav2 variants encoding aflibercept
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
EP4337267A1 (en) 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
KR20240035759A (ko) 2021-05-26 2024-03-18 스페어링비젼 막대세포-원추세포 이상증(rcd)에서 g-단백질-개폐-k+ 채널-매개 감광성 향상
AU2022281825A1 (en) 2021-05-28 2024-01-18 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
WO2022256561A1 (en) * 2021-06-03 2022-12-08 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US20250276088A1 (en) 2021-07-07 2025-09-04 Institut National de la Santé et de la Recherche Médicale Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
EP4373947A1 (en) 2021-07-19 2024-05-29 New York University Auf1 combination therapies for treatment of muscle degenerative disease
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
CR20240137A (es) 2021-08-20 2024-07-29 Biocad Joint Stock Co Método para obtener una cápside de virus adenoasociado modificada
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
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
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
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
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
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
WO2023108507A1 (en) * 2021-12-15 2023-06-22 National Institute Of Biological Sciences, Beijing Recombinant aav vectors and use thereof
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
JP2025506681A (ja) 2022-02-16 2025-03-13 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 組み換えアデノ随伴ウイルスによる血管新生ａｍｄにおけるｃｓｔ変動の低減方法
CN118715236A (zh) * 2022-02-17 2024-09-27 九天生物医药(上海)有限公司 具有经修饰的aav衣壳多肽的重组腺相关病毒
JP2025509328A (ja) 2022-03-07 2025-04-11 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 改良されたバッチａａｖ産生システムおよび方法
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
WO2023196873A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene forsuprachoidal 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
EP4508062A1 (en) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN116925192B (zh) * 2022-04-12 2025-04-29 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
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
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
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
EP4518973A1 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
EP4536694A1 (en) 2022-06-07 2025-04-16 Adverum Biotechnologies, Inc. Melanopsin variants for vision restoration
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
AU2023323390A1 (en) 2022-08-11 2025-02-13 Institute Of Molecular And Clinical Ophthalmology Basel (Iob) Human cone photoreceptor optogenetic constructs
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing 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
WO2024084075A1 (en) 2022-10-20 2024-04-25 Sparingvision Compositions and methods for treating retinal degenerative disorders
EP4357359A1 (en) 2022-10-20 2024-04-24 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
CN120112547A (zh) 2022-11-16 2025-06-06 瑞泽恩制药公司 包含膜结合il-12和蛋白酶可裂解接头的嵌合蛋白
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
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
IL321968A (en) 2023-02-02 2025-09-01 Quethera Ltd Recombinant adeno-associated virus vector
KR20250169640A (ko) 2023-03-10 2025-12-03 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
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
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
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
WO2024216244A2 (en) 2023-04-13 2024-10-17 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
WO2024229425A1 (en) * 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024240113A1 (zh) * 2023-05-23 2024-11-28 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
WO2025015317A2 (en) 2023-07-13 2025-01-16 Kriya Therapeutics, Inc. Complement pathway inhibitor fusions and methods of using the same
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
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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
WO2025108407A2 (en) 2023-11-23 2025-05-30 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
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070020624A1 (en) 1998-02-18 2007-01-25 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Pseudomonas aeruginosa for diagnostics and therapeutics
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same

Family Cites Families (101)

* 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
AU1686997A (en) 1995-12-08 1997-06-27 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
EP0927044A4 (en) 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET 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
CA2303768C (en) 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
EP1690868A1 (en) 1997-10-31 2006-08-16 Maxygen, Inc. 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
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
ATE294874T1 (de) * 1998-09-11 2005-05-15 Univ California Rekombinante adenovirus für gewebespezifische genexpression ins herz
DK1127150T3 (da) 1998-11-05 2007-09-24 Univ Pennsylvania Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
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
IL154987A0 (en) * 2000-09-22 2003-10-31 Virxsys Conditionally replicating viral vectors and their use
US6962815B2 (en) * 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
US20030129203A1 (en) * 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
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
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
WO2003089612A2 (en) 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
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
CA2511665A1 (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
EP1625222A2 (en) * 2003-05-14 2006-02-15 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. 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
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
JP2007524622A (ja) 2003-06-23 2007-08-30 エイアンドジー ファーマスーティカルズ インコーポレイテッド 抗癌治療に対する腫瘍細胞の感受性を回復させてアポトーシスを誘導するための組成物及び方法
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
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
KR100550967B1 (ko) 2003-12-03 2006-02-13 주식회사 엘지생활건강 유중수형 화장료 조성물
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
CN101124328A (zh) 2004-12-15 2008-02-13 北卡罗来纳查佩尔山大学 嵌合载体
US20060211650A1 (en) 2004-12-16 2006-09-21 Forest Laboratories, Inc. Reducing carbohydrate derivatives of adamantane amines, and synthesis and methods of use thereof
US20100172871A1 (en) 2005-02-17 2010-07-08 Flannery John G Muller Cell Specific Gene Therapy
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
EP2465925A1 (en) 2005-07-22 2012-06-20 The Board Of Trustees Of The Leland Light-activated cation channel and uses thereof
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
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
CA2685900A1 (en) * 2006-05-04 2007-11-15 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
CA2655957C (en) * 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) 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
BRPI0810172A2 (pt) 2007-04-13 2014-10-14 Catalyst Biosciences Inc Polipeptídeos de fator vii modificado e seus usos
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
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
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
CN103025344B (zh) 2010-05-17 2016-06-29 桑格摩生物科学股份有限公司 新型dna-结合蛋白及其用途
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
JP6042825B2 (ja) * 2011-02-10 2016-12-14 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
LT3254703T (lt) 2011-04-22 2020-05-25 The Regents Of The University Of California Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai
EP2748185A1 (en) 2011-08-24 2014-07-02 The Board of Trustees of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
TWI775096B (zh) 2012-05-15 2022-08-21 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)
JP6363958B2 (ja) 2012-12-25 2018-07-25 タカラバイオ株式会社 Ａａｖ変異体
DK2954051T3 (da) 2013-02-08 2019-07-08 Univ Pennsylvania Modificeret kapsid til genoverførsel til behandling af nethinden
JP6670743B2 (ja) 2013-05-29 2020-03-25 セレクティスＣｅｌｌｅｃｔｉｓ Ｉｉ型ｃｒｉｓｐｒシステムにおける新規のコンパクトなｃａｓ９足場
WO2014194132A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
NZ758025A (en) 2013-10-11 2022-07-01 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
CA2951707A1 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
HRP20201225T1 (hr) 2015-03-06 2020-11-13 Massachusetts Eye & Ear Infirmary Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
MX2018013463A (es) 2016-05-13 2019-07-04 4D Molecular Therapeutics Inc Capsides variantes de virus adenoasociados y metodos de uso de las mismas.
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CN110461368A (zh) * 2017-06-30 2019-11-15 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及其使用方法
EP3676373A4 (en) 2017-08-28 2021-06-09 The Regents of The University of California Adeno-associated virus capsid variants and methods of use thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070020624A1 (en) 1998-02-18 2007-01-25 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Pseudomonas aeruginosa for diagnostics and therapeutics
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GENE THERAPY. 2009, vol. 16, no. 7, pages 916-926.
NATURE BIOTECHNOLOGY. 2001, vol. 28, no. 1, pages 79-80.

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