CN117363655A - 增强腺相关病毒载体功能的方法 - Google Patents

增强腺相关病毒载体功能的方法 Download PDF

Info

Publication number
CN117363655A
CN117363655A CN202311294343.9A CN202311294343A CN117363655A CN 117363655 A CN117363655 A CN 117363655A CN 202311294343 A CN202311294343 A CN 202311294343A CN 117363655 A CN117363655 A CN 117363655A
Authority
CN
China
Prior art keywords
aav
amino acid
acid sequence
seq
sequences
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202311294343.9A
Other languages
English (en)
Chinese (zh)
Inventor
L·范登伯格
G·高
J·M·威尔森
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=36968964&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN117363655(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of CN117363655A publication Critical patent/CN117363655A/zh
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Manufacturing & Machinery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202311294343.9A 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法 Pending CN117363655A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US66908305P 2005-04-07 2005-04-07
US60/669,083 2005-04-07
US73349705P 2005-11-04 2005-11-04
US60/733,497 2005-11-04
PCT/US2006/013375 WO2006110689A2 (en) 2005-04-07 2006-04-07 Method of increasing the function of an aav vector
CN2006800105666A CN101203613B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN2006800105666A Division CN101203613B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法

Publications (1)

Publication Number Publication Date
CN117363655A true CN117363655A (zh) 2024-01-09

Family

ID=36968964

Family Applications (6)

Application Number Title Priority Date Filing Date
CN201611102646.6A Active CN106906241B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN202010980343.4A Pending CN112029737A (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN201210422034.0A Active CN102994549B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN202311294343.9A Pending CN117363655A (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN2006800105666A Active CN101203613B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN201410499227.5A Active CN104293835B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法

Family Applications Before (3)

Application Number Title Priority Date Filing Date
CN201611102646.6A Active CN106906241B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN202010980343.4A Pending CN112029737A (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN201210422034.0A Active CN102994549B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
CN2006800105666A Active CN101203613B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法
CN201410499227.5A Active CN104293835B (zh) 2005-04-07 2006-04-07 增强腺相关病毒载体功能的方法

Country Status (8)

Country Link
US (15) US8999678B2 (enExample)
EP (12) EP2359865B1 (enExample)
JP (5) JP5702519B2 (enExample)
CN (6) CN106906241B (enExample)
DE (2) DE23193601T1 (enExample)
DK (2) DK2359867T3 (enExample)
ES (6) ES2525067T3 (enExample)
WO (1) WO2006110689A2 (enExample)

Families Citing this family (410)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
EP2037892B1 (en) * 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
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
US20120093772A1 (en) 2008-05-20 2012-04-19 Alan Horsager Vectors for delivery of light sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
WO2010127097A1 (en) * 2009-04-30 2010-11-04 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
SG183929A1 (en) 2010-03-29 2012-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9102949B2 (en) 2010-04-23 2015-08-11 University Of Massachusetts CNS targeting AAV vectors and methods of use thereof
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
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
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
DK2699270T3 (en) 2011-04-22 2017-10-02 Univ California ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
CA2864879C (en) * 2012-02-17 2021-07-20 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
ES2862912T3 (es) * 2012-04-18 2021-10-08 Childrens Hospital Philadelphia Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
TWI702955B (zh) 2012-05-15 2020-09-01 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)
PT2879719T (pt) 2012-08-01 2018-10-08 Ohio State Innovation Foundation Administração intratecal do vírus adeno-associado 9 recombinante
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
HRP20201063T1 (hr) 2013-03-15 2020-11-27 The Trustees Of The University Of Pennsylvania Pripravci za liječenje mpsi
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CN105408486B (zh) * 2013-05-21 2020-07-14 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3054995A1 (en) * 2013-10-07 2016-08-17 Kiromic, LLC Compositions and methods for treating cardiovascular diseases using disease-specific promoter
CN115141258B (zh) 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
CA2939950C (en) 2014-03-09 2023-08-22 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
JP6689249B2 (ja) 2014-03-17 2020-05-20 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 錐体細胞における増強された遺伝子発現のための組成物および方法
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
JP6741590B2 (ja) 2014-04-25 2020-08-19 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 コレステロールレベルを低下させるためのldlr変異体および組成物中でのそれらの使用
JP6983511B2 (ja) 2014-04-25 2021-12-17 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 脳中の転移乳癌および他の癌を処置するための方法および組成物
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
PT3142750T (pt) 2014-05-13 2020-09-22 Univ Pennsylvania Composições que compreendem aav expressando construções de anticorpos duplos e suas utilizações
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
ES2993915T3 (en) * 2014-08-13 2025-01-13 Childrens Hospital Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
CA2964272A1 (en) 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
RU2716422C2 (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
KR20240063169A (ko) 2014-11-21 2024-05-10 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 중추 신경계에 표적화된 aav 벡터
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
PT3268024T (pt) 2015-03-10 2020-12-09 Univ Columbia Construtos de vetor viral recombinante glut1 adeno associado e métodos relacionados para restaurar a expressão de glut1
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2016160761A2 (en) * 2015-03-30 2016-10-06 Boehringer Ingelheim Vetmedica, Inc. Pcv2 orf2 carrier platform
WO2016176191A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
CA2985223A1 (en) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
MA48852A (fr) 2015-05-13 2020-04-01 Univ Pennsylvania Expression médiée par aav d'anticorps anti-grippaux et leurs procédés d'utilisation
IL256517B2 (en) 2015-06-23 2024-06-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
JP7166168B2 (ja) 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
IL257105B (en) 2015-07-31 2022-09-01 Univ Minnesota Modified cells and methods of therapy
CN108368521A (zh) 2015-08-06 2018-08-03 宾夕法尼亚州大学信托人 Glp-1和其在用于治疗代谢疾病的组合物中的用途
EA201890565A1 (ru) 2015-08-25 2019-04-30 Дьюк Юниверсити Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз
HK1257024A1 (zh) 2015-08-31 2019-10-11 宾夕法尼亚大学 用於治疗伴侣动物的aav-epo
IL258024B2 (en) 2015-09-24 2023-03-01 Univ Pennsylvania A preparation and method for the treatment of a complement-mediated disease
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
US12188037B2 (en) * 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
US11135313B2 (en) * 2015-10-28 2021-10-05 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
KR102794870B1 (ko) 2015-12-11 2025-04-10 매사추세츠 아이 앤드 이어 인퍼머리 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법
MX2018007080A (es) 2015-12-11 2018-11-12 Univ Pennsylvania Terapia genica para tratar hipercolesterolemia familiar.
CA3007330A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
EP3795180B1 (en) 2015-12-14 2025-11-05 The Trustees of The University of Pennsylvania Gene therapy for ocular disorders
US11124546B2 (en) * 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
SG11201808176TA (en) 2016-04-15 2018-10-30 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
IL262207B1 (en) 2016-04-15 2025-09-01 Univ Pennsylvania Compositions for treatment of wet age-related macular degeneration
SG11201808812RA (en) 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
DK4206216T3 (da) 2016-05-13 2025-12-01 4D Molecular Therapeutics Inc Adeno-associerede virusvarianter og fremgangsmåder til brug deraf
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
CA3252099A1 (en) 2016-05-18 2025-06-05 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
US11504435B2 (en) 2016-06-13 2022-11-22 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
IL264070B2 (en) 2016-07-08 2023-03-01 Univ Pennsylvania Methods and preparations for the treatment of disorders and diseases involving rdh12
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
MX2019000962A (es) * 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
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
JP2019524162A (ja) 2016-08-18 2019-09-05 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 モジュラーAAV送達システムによるCRISPR−Casゲノム編集
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3518985A4 (en) * 2016-09-29 2020-08-05 University of Florida Research Foundation, Incorporated AAVRH.10 VARIANTS WITH HOST ANTIBODY EXHAUST CAPACITIES AND MODIFIED TISSUE TARGETING PROPERTIES
WO2018071831A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
JP7181862B2 (ja) 2016-10-18 2022-12-01 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 腫瘍浸潤リンパ球および治療の方法
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018100054A1 (en) 2016-12-01 2018-06-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
CN110462037A (zh) * 2017-01-30 2019-11-15 学校法人日本医科大学 腺相关病毒(aav)衣壳蛋白的突变体
JP2020510648A (ja) 2017-02-20 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を処置するための遺伝子治療
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
WO2018160573A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018160582A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
EP4656244A2 (en) 2017-03-01 2025-12-03 The Trustees of the University of Pennsylvania Gene therapy for ocular disorders
TW202532642A (zh) 2017-04-14 2025-08-16 美商銳進科斯生物股份有限公司 使用由人類神經或膠細胞產生的重組人類艾杜糖醛酸鹽 (iduronate)-2-硫酸酯酶 (ids) 之黏多醣病ii之治療
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
WO2018209154A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
CN108103082B (zh) * 2017-05-11 2021-07-13 北京锦篮基因科技有限公司 一种腺相关病毒介导的lcat基因表达载体及其用途
US11591614B2 (en) 2017-05-11 2023-02-28 The Trustees Of The University Of Pennsylvania Gene therapy for ceroid lipofuscinoses
US11793887B2 (en) 2017-05-31 2023-10-24 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
WO2018232149A1 (en) 2017-06-14 2018-12-20 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019006182A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF
JP2020530307A (ja) * 2017-06-30 2020-10-22 インティマ・バイオサイエンス,インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
BR112020000063A2 (pt) 2017-07-06 2020-07-14 The Trustees Of The University Of Pennsylvania terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
KR102850887B1 (ko) 2017-08-03 2025-08-28 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
AU2018320849A1 (en) * 2017-08-25 2020-03-05 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
CA3059995A1 (en) 2017-08-28 2019-03-07 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
NZ763018A (en) 2017-09-20 2023-02-24 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
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.
CN119242711A (zh) 2017-10-16 2025-01-03 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079494A1 (en) 2017-10-18 2019-04-25 Regenxbio, Inc. TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION
BR112020007405A2 (pt) 2017-10-18 2020-12-08 Regenxbio Inc. Produtos terapêuticos de anticorpo pós-translacionalmente modificados totalmente humanos
PT3717636T (pt) 2017-11-27 2023-05-30 4D Molecular Therapeutics Inc Cápsides variantes de vírus adeno-associado e utilização para inibir a angiogénese
AU2018375163A1 (en) 2017-11-30 2020-06-11 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIB
JP7389744B2 (ja) 2017-11-30 2023-11-30 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 ムコ多糖症iiia型のための遺伝子療法
MY207644A (en) 2017-12-19 2025-03-07 Akouos Inc Aav-mediated delivery of therapeutic antibodies to the inner ear
US10806802B2 (en) 2018-01-18 2020-10-20 University Of Guelph Adeno-associated virus particle with mutated capsid and methods of use thereof
AU2019211465B2 (en) 2018-01-23 2022-07-21 Mount Spec Investments Pty Ltd Expression vector and method
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
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
CN112352050A (zh) * 2018-02-27 2021-02-09 宾夕法尼亚州大学信托人 新颖腺相关病毒(aav)载体、具有降低的衣壳脱酰胺化的aav载体及其用途
EP3768695A4 (en) * 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
CN112218880A (zh) * 2018-03-29 2021-01-12 阿斯克肋匹奥生物制药公司 逃避中和的肝趋向性重组aav6载体
EP3775266A4 (en) 2018-04-05 2021-06-30 Massachusetts Eye and Ear Infirmary METHOD OF MANUFACTURING AND USING COMBINATIONAL BAR-CODED NUCLEIC ACID LIBRARIES WITH DEFINED VARIATION
AU2019249890B2 (en) 2018-04-05 2024-10-10 Association Institut De Myologie Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
JP7575273B2 (ja) 2018-04-16 2024-10-29 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 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
KR20210009317A (ko) 2018-04-18 2021-01-26 더 트러스티스 오브 콜롬비아 유니버시티 인 더 시티 오브 뉴욕 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 초래된 질환에 대한 유전자 요법
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
US20210231560A1 (en) 2018-04-29 2021-07-29 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
US20210207168A1 (en) 2018-05-08 2021-07-08 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3806888B1 (en) 2018-06-12 2024-01-31 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
US12070702B2 (en) 2018-06-14 2024-08-27 Regenxbio Inc. Anion exchange chromatography for recombinant AAV production
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
WO2020016655A2 (en) 2018-07-17 2020-01-23 Helixmith Co., Ltd. Treatment of neuropathy with dna constructs expressing igf-1 isoforms
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
BR112021001878A2 (pt) * 2018-07-31 2021-04-27 Cornell University métodos de terapia genética para o controle da função do órgão
JP2021533757A (ja) 2018-08-10 2021-12-09 リジェネクスバイオ インコーポレイテッド 組換えaav生成のためのスケーラブルな方法
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
EP3841109A4 (en) * 2018-08-21 2022-09-21 Massachusetts Eye and Ear Infirmary COMPOSITIONS AND METHODS TO MODULATE THE TRANSDUCTION EFFICIENCY OF ADENO-ASSOCIATED VIRUSES
CA3111076A1 (en) 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ascl1
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
MX2021003869A (es) 2018-10-01 2021-09-08 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
US12472268B2 (en) 2018-10-01 2025-11-18 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
CA3115248A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
CA3116701A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
EP3867412A1 (en) 2018-10-15 2021-08-25 REGENXBIO Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
WO2020117898A1 (en) 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
TWI848038B (zh) 2019-01-04 2024-07-11 美商奧崔基尼克斯製藥公司 用於治療威爾森氏症之基因療法構築體
AU2020208467A1 (en) 2019-01-18 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing AAV particles
CA3125294A1 (en) * 2019-01-23 2020-07-30 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
CN119390787A (zh) 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
TW202045728A (zh) 2019-02-26 2020-12-16 賓州大學委員會 用於治療克拉培氏病之組成物
US20220154217A1 (en) 2019-04-01 2022-05-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN113966236A (zh) 2019-04-03 2022-01-21 再生生物股份有限公司 眼睛病状的基因疗法
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
PL3953483T3 (pl) 2019-04-11 2024-04-22 Regenxbio Inc. Sposoby chromatografii wykluczania do charakteryzowania kompozycji rekombinowanych wirusów towarzyszących adenowirusom
MX2021012564A (es) 2019-04-19 2022-01-18 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados.
CN114127088B (zh) * 2019-04-23 2024-05-14 国家医疗保健研究所 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物
SG11202111414RA (en) 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
WO2020222858A1 (en) * 2019-04-27 2020-11-05 Ocugen, Inc. Adeno-associated virus vector mediated gene therapy for ophthalmic diseases
BR112021021502A2 (pt) * 2019-04-29 2022-07-19 Univ Pennsylvania Capsídeos de aav e composições contendo os mesmos
MX2021013420A (es) 2019-05-03 2022-02-03 Univ Pennsylvania Composiciones utiles en el tratamiento de leucodistrofia metacromatica.
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US11319354B2 (en) 2019-07-10 2022-05-03 Masonic Medical Research Laboratory VGLL4 with UCP-1 cis-regulatory element and method of use thereof
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
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
RS66255B1 (sr) * 2019-07-15 2024-12-31 Meiragtx Uk Ii Ltd Modifikovani proteini aav kapsida za lečenje artritične bolesti
WO2021021674A1 (en) 2019-07-26 2021-02-04 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
AU2020336314A1 (en) 2019-08-26 2022-04-07 Regenxbio Inc. Treatment of diabetic retinopathy with fully-human post-translationally modified anti-VEGF Fab
US20230092895A1 (en) 2019-08-30 2023-03-23 Obsidian Therapeutics, Inc. Tandem cd19 car-based compositions and methods for immunotherapy
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021046451A1 (en) 2019-09-06 2021-03-11 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
KR20220079857A (ko) 2019-09-13 2022-06-14 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 Aav-상용성 라미닌-링커 중합 단백질
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
JP2022552262A (ja) 2019-10-07 2022-12-15 リジェネックスバイオ インコーポレイテッド アデノ随伴ウイルスベクター医薬組成物および方法
CN113518824B (zh) * 2019-10-16 2024-02-23 上海药明康德新药开发有限公司 新的aav变体
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
BR112022009895A2 (pt) 2019-11-28 2022-08-09 Regenxbio Inc Construtos de terapia de gene de microdistrofina e usos dos mesmos
AU2020398904A1 (en) * 2019-12-10 2022-06-30 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
US11981912B2 (en) 2019-12-10 2024-05-14 Takeda Pharma ceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
CA3166374A1 (en) 2019-12-31 2021-07-08 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
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
BR112022014563A2 (pt) 2020-01-22 2022-09-13 Regenxbio Inc Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i)
AR121201A1 (es) 2020-01-29 2022-04-27 Regenxbio Inc Tratamiento de mucopolisacaridosis iva
CA3167741A1 (en) 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
US20230190966A1 (en) 2020-02-02 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CA3164714A1 (en) 2020-02-14 2021-08-19 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
JP2023518327A (ja) * 2020-02-25 2023-04-28 チルドレンズ・メディカル・リサーチ・インスティテュート アデノ随伴ウイルスカプシドポリペプチドおよびベクター
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
BR112022019047A2 (pt) 2020-03-27 2022-11-01 UCB Biopharma SRL Peptídeos de domínio knob autônomos
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
KR20220167380A (ko) * 2020-04-13 2022-12-20 매사추세츠 아이 앤드 이어 인퍼머리 코로나바이러스에 대한 백신을 제조 및 사용하는 방법
IL297200A (en) 2020-04-15 2022-12-01 Voyager Therapeutics Inc tau binding compounds
CN116057175A (zh) 2020-05-12 2023-05-02 宾夕法尼亚州大学信托人 可用于治疗克拉伯病的组合物
KR20230010670A (ko) 2020-05-12 2023-01-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
WO2021231538A2 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
CN116801911A (zh) 2020-05-13 2023-09-22 阿库斯股份有限公司 用于治疗slc26a4相关听力损失的组合物和方法
AU2021282257A1 (en) 2020-05-26 2022-12-01 Shape Therapeutics Inc. High throughput engineering of functional 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
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
EP4171738A1 (en) 2020-06-17 2023-05-03 The Trustees of The University of Pennsylvania Compositions and methods for treatment of gene therapy patients
US20230242905A1 (en) * 2020-07-03 2023-08-03 Genethon Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
EP4179091A1 (en) 2020-07-10 2023-05-17 Institut National De La Sante Et De La Recherche Medicale - Inserm Methods and compositions for treating epilepsy
CN116438311A (zh) 2020-07-13 2023-07-14 宾夕法尼亚州大学信托人 可用于治疗夏科-马里-图思病的组合物
WO2022026409A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CN115927398B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
CN116323953A (zh) 2020-07-30 2023-06-23 沙普治疗股份有限公司 诱导产生raav病毒粒子的稳定细胞系
EP4200429A1 (en) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
US20230364264A1 (en) 2020-08-26 2023-11-16 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
US20230383278A1 (en) 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
CA3194861A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
US20240024508A1 (en) 2020-10-07 2024-01-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
IL301643A (en) 2020-10-07 2023-05-01 Regenxbio Inc Gene therapy for ocular manifestations of CLN2 disease
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
CA3193697A1 (en) 2020-10-07 2022-04-14 Joseph Bruder Adeno-associated viruses for ocular delivery of gene therapy
CA3193833A1 (en) 2020-10-09 2022-04-14 Juliette HORDEAUX Compositions and methods for treatment of fabry disease
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
AR123948A1 (es) 2020-10-28 2023-01-25 Regenxbio Inc ANTICUERPOS ANTI-TNF-a VECTORIZADOS PARA INDICACIONES OCULARES
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP4237453A1 (en) 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized tnf-alpha antagonists for ocular indications
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
MX2023006445A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
WO2022119839A1 (en) 2020-12-01 2022-06-09 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
KR20230120128A (ko) 2020-12-16 2023-08-16 리젠엑스바이오 인크. 재조합 바이러스 입자의 생산 방법
US20240139344A1 (en) 2020-12-29 2024-05-02 Akouos, Inc. Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
WO2022147087A1 (en) 2020-12-29 2022-07-07 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
EP4277933A4 (en) 2021-01-14 2024-12-11 Senti Biosciences, Inc. SECRETABLE PAYLOAD REGULATION
CA3205209A1 (en) 2021-01-21 2022-07-28 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US11926842B2 (en) 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
EP4291249A2 (en) 2021-02-10 2023-12-20 RegenxBio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
TW202302855A (zh) 2021-02-26 2023-01-16 日商武田藥品工業股份有限公司 用於治療法布瑞氏症之組成物及方法
AU2022258312A1 (en) 2021-04-12 2023-10-26 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
MX2023012511A (es) 2021-04-23 2024-01-08 Univ Rochester Edición del genóma por inserción no homóloga de adn dirigida utilizando una proteína de fusión retroviral de integrasa-cas y métodos de tratamiento.
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
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
WO2022272297A1 (en) 2021-06-25 2022-12-29 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
EP4373947A1 (en) 2021-07-19 2024-05-29 New York University Auf1 combination therapies for treatment of muscle degenerative disease
JP2024534771A (ja) 2021-08-11 2024-09-26 サナ バイオテクノロジー,インコーポレイテッド 補体媒介性炎症反応を低減するための同種異系細胞療法のための遺伝子改変細胞
AU2022326565A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
WO2023019225A2 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
MX2024001208A (es) 2021-08-11 2024-04-22 Sana Biotechnology Inc Celulas primarias geneticamente modificadas para terapia celular alogenica.
US20250019721A1 (en) 2021-08-11 2025-01-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
EP4392016A1 (en) 2021-08-24 2024-07-03 Oxford Biomedica (US) LLC Adeno-associated virus formulations
CN118159663A (zh) * 2021-08-25 2024-06-07 北海康成制药有限公司 包含嗜肝衣壳蛋白和酸性α-葡糖苷酶(GAA)的AAV颗粒及其治疗庞贝病的用途
WO2023034880A2 (en) 2021-08-31 2023-03-09 Scout Bio, Inc. Antigen-binding molecules and uses thereof
US20250288692A1 (en) 2021-09-30 2025-09-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
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
WO2023060272A2 (en) 2021-10-07 2023-04-13 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
WO2023072181A1 (en) * 2021-10-28 2023-05-04 Shanghai Vitalgen Biopharma Co., Ltd. Adeno-associated virus capsid
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
US20230183658A1 (en) 2021-10-29 2023-06-15 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250051750A1 (en) 2021-11-29 2025-02-13 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
WO2023102078A2 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
JP2024543211A (ja) 2021-12-01 2024-11-19 シェイプ セラピューティクス インコーポレイテッド 全身投与用の機能的aavカプシド
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CN118510905A (zh) * 2021-12-06 2024-08-16 学校法人顺天堂 改变型腺伴随病毒载体
WO2023114901A2 (en) 2021-12-15 2023-06-22 Oxford Biomedica Solutions Llc Methods and compositions for the production of adeno-associated virus
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
MX2024009186A (es) 2022-01-25 2024-09-30 Univ Pennsylvania Cápsides de aav para mejorar la transducción cardiaca y desdireccionamiento del hígado.
EP4473097A1 (en) 2022-02-02 2024-12-11 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
AU2023215253A1 (en) 2022-02-02 2024-09-19 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
AU2023220128A1 (en) 2022-02-17 2024-08-22 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
EP4490278A1 (en) 2022-03-07 2025-01-15 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
EP4499154A1 (en) 2022-03-25 2025-02-05 REGENXBIO Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
US20250213730A1 (en) 2022-04-01 2025-07-03 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
EP4504149A1 (en) 2022-04-06 2025-02-12 RegenxBio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoidal 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
CN119317645A (zh) 2022-04-06 2025-01-14 宾夕法尼亚州大学信托人 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法
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
EP4507741A1 (en) 2022-04-14 2025-02-19 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
US20250288697A1 (en) 2022-05-03 2025-09-18 Regenxbio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
AR129215A1 (es) 2022-05-03 2024-07-31 Regenxbio Inc Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos
JP2025517628A (ja) 2022-05-06 2025-06-10 ノバルティス アーゲー 新規の組換えaav vp2融合ポリペプチド
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
CA3259982A1 (en) 2022-06-30 2024-01-04 Indapta Therapeutics, Inc. COMBINATION OF MODIFIED NATURAL KILLER (NK) CELLS AND ANTIBODY THERAPY AND ASSOCIATED METHODS
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
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
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
JP2025529884A (ja) 2022-08-25 2025-09-09 武田薬品工業株式会社 ファブリー病の治療に使用するための組成物
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
CN115960177B (zh) * 2022-10-09 2023-07-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
EP4601710A2 (en) 2022-10-11 2025-08-20 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
CN117304256B (zh) * 2022-12-08 2024-04-16 广州派真生物技术有限公司 腺相关病毒突变体及其应用
AU2023398931A1 (en) 2022-12-17 2025-07-10 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
EP4646478A1 (en) 2023-01-06 2025-11-12 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
US20240401025A1 (en) 2023-05-03 2024-12-05 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
WO2024229302A1 (en) 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
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
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
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
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
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
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
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
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
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
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
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
CN118460614B (zh) * 2024-07-05 2025-03-11 凌意(杭州)生物科技有限公司 一种腺相关病毒Cap蛋白的表达框
CN118754952B (zh) * 2024-09-06 2025-01-10 上海玮美基因科技有限责任公司 一种新型腺相关病毒及其应用

Family Cites Families (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
EP0162067B1 (en) 1983-10-28 1992-07-15 Genetics Institute, Inc. Production of factor viii and related products
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
FI86885C (fi) 1984-04-20 1992-10-26 Genentech Inc Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
KR927003812A (ko) 1989-11-17 1992-12-18 노보 노르디스크 아크티에 셀스카브 인자 viii:c 활성을 갖는 단백질 복합체 및 그의 제조
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US6174666B1 (en) 1992-03-27 2001-01-16 The United States Of America As Represented By The Department Of Health And Human Services Method of eliminating inhibitory/instability regions from mRNA
WO1994011013A1 (en) 1992-11-13 1994-05-26 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
ATE272122T1 (de) 1993-06-10 2004-08-15 Bayer Ag Vektoren und zelllinien von säugetieren mit erhöhter produktivität
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
WO1997003195A1 (en) 1995-07-11 1997-01-30 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services 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
JP2001517454A (ja) 1997-09-19 2001-10-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
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
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
EP1218527A2 (en) * 1999-07-02 2002-07-03 Onyx Pharmaceuticals, Inc. Adenoviral vectors for treating disease
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
CA2373110A1 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
CA2403817C (en) * 2000-03-31 2012-05-29 The General Hospital Corporation Methods of modulating hair growth
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
JP2004534543A (ja) * 2001-07-13 2004-11-18 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン シュードタイプ化アデノ関連ウイルスおよびその使用
US6410330B1 (en) 2001-07-27 2002-06-25 Coulter International Corp. Method for measurement of nucleated red blood cells
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
NZ618298A (en) * 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US7235393B2 (en) 2002-04-29 2007-06-26 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
CA2499824C (en) 2002-09-30 2009-12-08 Canon Kabushiki Kaisha Liquid supply system, fluid communicating structure, ink supply system, and inkjet recording head utilizing the fluid communicating structure
JP2004157072A (ja) 2002-11-08 2004-06-03 Japan Science & Technology Agency 免疫反応測定に用いられる高感度磁性マーカー
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
PT2657248T (pt) * 2003-06-19 2017-06-26 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
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
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006119432A2 (en) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
CN101495624A (zh) 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
ES2862912T3 (es) * 2012-04-18 2021-10-08 Childrens Hospital Philadelphia Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
CN115141258B (zh) * 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
IL256517B2 (en) 2015-06-23 2024-06-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
JP7166168B2 (ja) * 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
CN116096734A (zh) * 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向

Also Published As

Publication number Publication date
JP5677681B2 (ja) 2015-02-25
US8999678B2 (en) 2015-04-07
JP5702519B2 (ja) 2015-04-15
EP1866422A2 (en) 2007-12-19
CN102994549B (zh) 2015-02-11
CN104293835A (zh) 2015-01-21
WO2006110689A2 (en) 2006-10-19
ES2434723T3 (es) 2013-12-17
US20200208176A1 (en) 2020-07-02
EP3409296A1 (en) 2018-12-05
CN106906241B (zh) 2020-09-11
EP4282956A3 (en) 2024-03-13
CN101203613A (zh) 2008-06-18
JP2012165744A (ja) 2012-09-06
JP2016168047A (ja) 2016-09-23
CN104293835B (zh) 2017-07-04
EP2359867B1 (en) 2014-10-08
EP2359866A1 (en) 2011-08-24
EP4282957A2 (en) 2023-11-29
ES2525067T3 (es) 2014-12-17
US20250179525A1 (en) 2025-06-05
US10626415B2 (en) 2020-04-21
US20180057841A1 (en) 2018-03-01
JP2014204723A (ja) 2014-10-30
US20170191079A1 (en) 2017-07-06
EP3603676A1 (en) 2020-02-05
CN101203613B (zh) 2012-12-12
CN112029737A (zh) 2020-12-04
CN106906241A (zh) 2017-06-30
JP5912147B2 (ja) 2016-04-27
US20250171801A1 (en) 2025-05-29
EP2359867A1 (en) 2011-08-24
ES2525143T3 (es) 2014-12-18
EP4234687A2 (en) 2023-08-30
CN102994549A (zh) 2013-03-27
JP6275769B2 (ja) 2018-02-07
US20240117378A1 (en) 2024-04-11
US10947561B2 (en) 2021-03-16
WO2006110689A3 (en) 2007-04-05
EP2357010B1 (en) 2013-06-12
US20230416780A1 (en) 2023-12-28
EP1866422B1 (en) 2016-04-06
EP2357010A1 (en) 2011-08-17
EP4282956A2 (en) 2023-11-29
DK2359867T3 (en) 2015-01-05
DE23193606T1 (de) 2024-02-15
EP2383346A1 (en) 2011-11-02
DE23193601T1 (de) 2024-02-15
ES2580044T3 (es) 2016-08-18
EP2383346B1 (en) 2014-10-08
JP2008538286A (ja) 2008-10-23
EP3085389A1 (en) 2016-10-26
EP4282957A3 (en) 2024-03-13
ES2428218T3 (es) 2013-11-06
EP3603676A8 (en) 2020-03-25
US20230287452A1 (en) 2023-09-14
US11680274B2 (en) 2023-06-20
US20230272424A1 (en) 2023-08-31
US20210040504A1 (en) 2021-02-11
US10301648B2 (en) 2019-05-28
EP2359865B1 (en) 2013-10-02
US20190218574A1 (en) 2019-07-18
JP2015096070A (ja) 2015-05-21
EP2359866B1 (en) 2013-07-17
JP6039632B2 (ja) 2016-12-07
US20150159173A1 (en) 2015-06-11
US20200325491A1 (en) 2020-10-15
US20230340532A1 (en) 2023-10-26
US20090197338A1 (en) 2009-08-06
EP2359865A1 (en) 2011-08-24
ES2426245T3 (es) 2013-10-22
DK2383346T3 (en) 2015-01-05

Similar Documents

Publication Publication Date Title
US20230272424A1 (en) Method of increasing the function of an aav vector
HK40042489A (en) Method of increasing the function of an aav vector

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination