DE23193606T1 - Verfahren zur erhöhung der funktion eines aav-vektors - Google Patents

Verfahren zur erhöhung der funktion eines aav-vektors Download PDF

Info

Publication number
DE23193606T1
DE23193606T1 DE23193606.3T DE23193606T DE23193606T1 DE 23193606 T1 DE23193606 T1 DE 23193606T1 DE 23193606 T DE23193606 T DE 23193606T DE 23193606 T1 DE23193606 T1 DE 23193606T1
Authority
DE
Germany
Prior art keywords
aav
seq
proteins
amino acids
capsid
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
DE23193606.3T
Other languages
German (de)
English (en)
Inventor
Luk Vandenberghe
Guangping Gao
James N. Wilson
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
Original Assignee
University of Pennsylvania
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=DE23193606(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by University of Pennsylvania filed Critical University of Pennsylvania
Publication of DE23193606T1 publication Critical patent/DE23193606T1/de
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
DE23193606.3T 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors Pending DE23193606T1 (de)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US66908305P 2005-04-07 2005-04-07
US669083P 2005-04-07
US73349705P 2005-11-04 2005-11-04
US733497P 2005-11-04
EP23193606.3A EP4282957A3 (en) 2005-04-07 2006-04-07 Method of increasing the function of an aav vector

Publications (1)

Publication Number Publication Date
DE23193606T1 true DE23193606T1 (de) 2024-02-15

Family

ID=36968964

Family Applications (2)

Application Number Title Priority Date Filing Date
DE23193606.3T Pending DE23193606T1 (de) 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors
DE23193601.4T Pending DE23193601T1 (de) 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors

Family Applications After (1)

Application Number Title Priority Date Filing Date
DE23193601.4T Pending DE23193601T1 (de) 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors

Country Status (8)

Country Link
US (15) US8999678B2 (enExample)
EP (12) EP1866422B1 (enExample)
JP (5) JP5702519B2 (enExample)
CN (6) CN102994549B (enExample)
DE (2) DE23193606T1 (enExample)
DK (2) DK2383346T3 (enExample)
ES (6) ES2434723T3 (enExample)
WO (1) WO2006110689A2 (enExample)

Families Citing this family (445)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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
EP1668143B1 (en) * 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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
CN101511373B (zh) * 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
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
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
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
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
DK2425000T3 (da) * 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma 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
US10738326B2 (en) * 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
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
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
CA2880653C (en) 2012-08-01 2022-05-17 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
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
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
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
KR102298579B1 (ko) * 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CN106029107A (zh) * 2013-10-07 2016-10-12 基罗米克有限责任公司 使用疾病特异性启动子治疗心血管疾病的组合物和方法
HUE052676T2 (hu) * 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
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
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
US11555059B2 (en) 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
CR20160519A (es) 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SG11201701033QA (en) * 2014-08-13 2017-03-30 Philadelphia Children Hospital An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma 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
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
MX2017011615A (es) 2015-03-10 2018-04-11 Univ Columbia Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1.
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US10555994B2 (en) * 2015-03-30 2020-02-11 Boehringer Ingelheim Animal Health USA Inc. PCV2 ORF2 carrier platform
JP6851319B2 (ja) 2015-04-27 2021-03-31 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 ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
HK1252512A1 (zh) 2015-05-07 2019-05-31 Massachusetts Eye & Ear Infirmary 向眼部遞送藥劑的方法
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
EP4378487A3 (en) 2015-06-23 2024-09-04 The Children's Hospital of 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
EP4134375A1 (en) 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof
EP4339287A3 (en) 2015-07-31 2024-05-22 Regents Of The University Of Minnesota Modified cells and methods of therapy
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
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
EP3359663A4 (en) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
WO2017062750A1 (en) 2015-10-09 2017-04-13 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
WO2017070476A2 (en) * 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using 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
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
EP4085934A1 (en) 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP3387138B2 (en) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
CA3007476A1 (en) 2015-12-11 2017-06-15 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
CN109069668B (zh) 2015-12-14 2023-04-18 宾夕法尼亚州大学信托人 用于眼病的基因疗法
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
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
US11253612B2 (en) 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
IL262207B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP2019517274A (ja) 2016-06-13 2019-06-24 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ 最適化されたｃｌｎ１遺伝子および発現カセットおよびそれらの使用
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US11197936B2 (en) 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
JP6994018B2 (ja) * 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
CN109640949B (zh) 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
CA3034089A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
US11078238B2 (en) * 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
ES2931960T3 (es) 2016-12-01 2023-01-05 Inst Nat Sante Rech Med Composiciones farmacéuticas para el tratamiento de enfermedades degenerativas retinianas
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
JP2020508289A (ja) 2017-01-31 2020-03-19 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
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
TW201837170A (zh) 2017-02-28 2018-10-16 賓州大學委員會 新穎ａａｖ媒介的流感疫苗
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018160849A1 (en) 2017-03-01 2018-09-07 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
CN110913954A (zh) 2017-03-03 2020-03-24 黑曜石疗法公司 免疫疗法的组合物和方法
AU2018227583B2 (en) 2017-03-03 2023-06-01 Obsidian Therapeutics, Inc. CD19 compositions and methods for immunotherapy
KR20240093919A (ko) 2017-04-14 2024-06-24 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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
BR112019023303A2 (pt) 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais
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
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (ｆｇｆ21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
US11793887B2 (en) 2017-05-31 2023-10-24 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
US11827898B2 (en) 2017-06-14 2023-11-28 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
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
KR20250023600A (ko) 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
JP2020533968A (ja) * 2017-08-25 2020-11-26 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 組換えアデノ随伴ベクター
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
DK3684423T5 (da) * 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
EP3684938A1 (en) 2017-09-22 2020-07-29 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.
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MX2020003945A (es) 2017-10-18 2020-11-09 Regenxbio Inc Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente .
KR20200067195A (ko) 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
EA202091352A1 (ru) 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
US11555206B2 (en) 2017-11-30 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIA
CA3086045A1 (en) 2017-12-19 2019-06-27 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
US12305185B2 (en) 2018-01-23 2025-05-20 Mount Spec Investments Pty Ltd Expression vector and method
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
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
IL276859B2 (en) * 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
EP3781187A4 (en) * 2018-03-29 2021-12-22 Asklepios Biopharmaceutical, Inc. TROPICAL RECOMBINANT AAV6 LIVER VECTORS TO Bypass NEUTRALIZATION
WO2019195701A1 (en) 2018-04-05 2019-10-10 Massachusetts Eye And Ear Infirmary Methods of making and using combinatorial barcoded nucleic acid libraries having defined variation
JP7374119B2 (ja) 2018-04-05 2023-11-06 ジェネトン 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型
CA3099958A1 (en) 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
BR112020021324A2 (pt) 2018-04-18 2021-01-19 The Trustees Of Columbia University In The City Of New York Terapia gênica para doenças causadas por pools de nucleotídeos em desequilíbrio incluindo síndromes de depleção do dna mitocondrial
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
CA3098566A1 (en) 2018-04-29 2019-11-07 Zhuchun WU Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
KR102877876B1 (ko) 2018-05-08 2025-10-28 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 Aav-상용성 라미닌-링커 중합 단백질
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
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20220403001A1 (en) 2018-06-12 2022-12-22 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
IL319002A (en) 2018-06-14 2025-04-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
CA3106078A1 (en) 2018-07-17 2020-01-23 Junghun Lee 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
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
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
US12195745B2 (en) * 2018-08-21 2025-01-14 Massachusetts Eye And Ear Infirmary Compositions and methods for modulating transduction efficiency of adeno-associated viruses
KR20210102870A (ko) 2018-08-30 2021-08-20 테나야 테라퓨틱스, 인코포레이티드 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354296A1 (en) 2018-10-01 2021-04-29 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP3860659A1 (en) 2018-10-01 2021-08-11 The Trustees of the University of Pennsylvania Compositions useful for treating gm1 gangliosidosis
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 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
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
JP2022514112A (ja) 2018-10-15 2022-02-09 リジェネクスバイオ インコーポレイテッド 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
BR112021009913A2 (pt) 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica
KR20210112339A (ko) 2019-01-04 2021-09-14 울트라제닉스 파마수티컬 인코포레이티드 윌슨병을 치료하기 위한 유전자 요법 구축물
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2020154535A1 (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
MX2021010134A (es) 2019-02-22 2021-09-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
EP3931337A1 (en) 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
AU2020232692A1 (en) 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP4667578A3 (en) 2019-04-03 2026-04-01 REGENXBIO Inc. Gene therapy for eye pathologies
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
ES2965341T3 (es) 2019-04-11 2024-04-12 Regenxbio Inc Métodos de cromatografía de exclusión por tamaño para la caracterización de composiciones de virus adenoasociados recombinantes
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
JP7630443B2 (ja) 2019-04-19 2025-02-17 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
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
US20220195462A1 (en) 2019-04-24 2022-06-23 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
JP2022530845A (ja) * 2019-04-27 2022-07-04 オクジェン アイエヌシー． 眼疾患に対するａｄｅｎｏ随伴ウイルス連ウイルスベクター媒介遺伝子治療
WO2020223232A1 (en) * 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
JP7715640B2 (ja) 2019-05-03 2025-07-30 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 異染性白質ジストロフィーの治療に有用な組成物
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
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
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
SMT202400486T1 (it) * 2019-07-15 2025-01-14 Meiragtx Uk Ii Ltd Proteine di capside avv modificate per il trattamento della malattia artritica
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
EP4010357A2 (en) 2019-08-09 2022-06-15 University of Florida Research Foundation, Incorporated Aav capsid variants for targeting human glioblastoma cells
CA3149401A1 (en) 2019-08-26 2021-03-04 Stephen Joseph Pakola 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
WO2021046155A1 (en) 2019-09-03 2021-03-11 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
CN114555814A (zh) 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021071835A1 (en) 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
IL291944A (en) 2019-10-10 2022-06-01 Solid Biosciences Inc Modified aav capsids and uses thereof
WO2021073568A1 (en) * 2019-10-16 2021-04-22 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
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
US12636380B2 (en) 2019-11-22 2026-05-26 The University Of North Carolina At Chapel Hill Methods and compositions for increasing transduction efficiency with cell membrane fusion proteins
WO2021108467A1 (en) 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes and evade the humoral immune response
AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
KR20220112283A (ko) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
CA3161367A1 (en) * 2019-12-10 2021-06-17 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
BR112022011916A2 (pt) 2019-12-19 2022-09-06 Univ Pennsylvania Composições para tratar a ataxia de friedreich
CN116096904A (zh) 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
KR20220131522A (ko) 2020-01-22 2022-09-28 리젠엑스바이오 인크. 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료
BR112022015036A2 (pt) 2020-01-29 2022-10-11 Regenxbio Inc Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
US20230088992A1 (en) 2020-01-29 2023-03-23 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
WO2021155337A1 (en) 2020-02-02 2021-08-05 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
AU2021218703A1 (en) 2020-02-14 2022-07-28 Ultragenyx Pharmaceutical Inc. Gene therapy for treating CDKL5 deficiency disorder
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
JP2023519576A (ja) 2020-03-27 2023-05-11 ユーシービー バイオファルマ エスアールエル 自律ノブドメインペプチド
US12686874B2 (en) 2020-03-31 2026-07-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2021211629A2 (en) * 2020-04-13 2021-10-21 Massachusetts Eye And Ear Infirmary Methods of making and using a vaccine against coronavirus
CA3180222A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
AU2021271635A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4165194A2 (en) 2020-05-13 2023-04-19 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
KR20230041965A (ko) 2020-05-13 2023-03-27 아카우오스, 인크. Slc26a4-연관 청력손실을 치료하기 위한 조성물 및 방법
JP2023529315A (ja) 2020-05-26 2023-07-10 シェイプ セラピューティクス インコーポレイテッド 機能的ａａｖカプシドのハイスループット工学操作
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
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER 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
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
WO2022015715A1 (en) 2020-07-13 2022-01-20 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
WO2022020548A2 (en) 2020-07-23 2022-01-27 President And Fellows Of Harvard College Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
CN116121274B (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
US12054738B2 (en) 2020-07-30 2024-08-06 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
US20230372539A1 (en) 2020-08-24 2023-11-23 The Trustees Of The University Of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
AU2021332331A1 (en) 2020-08-26 2023-03-09 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
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
EP4225777A2 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Adeno-associated viruses for ocular delivery of gene therapy
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
TW202228648A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如高黏度調配物之用於脈絡膜上投與之調配物
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Ｃｌｎ２疾患の眼症状に対する遺伝子療法
TW202228646A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如凝膠調配物之用於脈絡膜上投與之調配物
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
WO2022082109A1 (en) 2020-10-18 2022-04-21 The Trustees Of The University Of Pennsylvania 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
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
KR20230098176A (ko) 2020-10-29 2023-07-03 리젠엑스바이오 인크. 안구 적응증을 위한 벡터화된 tnf-알파 길항제
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
WO2022119890A1 (en) 2020-12-01 2022-06-09 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
EP4263841A1 (en) 2020-12-16 2023-10-25 REGENXBIO Inc. Method of producing a recombinant adeno-associated virus particle
CN117157108A (zh) 2020-12-29 2023-12-01 阿库斯股份有限公司 用于治疗clrn1相关听力损失和/或视力损失的组合物和方法
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
WO2022155500A1 (en) 2021-01-14 2022-07-21 Senti Biosciences, Inc. Secretable payload regulation
AU2022210592A1 (en) 2021-01-21 2023-07-13 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
WO2022169774A1 (en) 2021-02-02 2022-08-11 University Of Massachusetts Inverted terminal repeats from aav serotypes 8 and rh.39 in gene therapy vectors
AU2022221284A1 (en) 2021-02-10 2023-08-17 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
AR124981A1 (es) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
IL307633A (en) 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (SBMA)
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022226296A2 (en) 2021-04-23 2022-10-27 University Of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
IL307902A (en) 2021-04-26 2023-12-01 Regenxbio Inc Microdystrophin gene therapy administration for treatment of dystrophinopathies
EP4334454A2 (en) 2021-05-04 2024-03-13 RegenxBio Inc. Novel aav vectors and methods and uses thereof
US20240358857A1 (en) 2021-05-11 2024-10-31 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
US20230055381A1 (en) 2021-06-25 2023-02-23 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
KR20240051112A (ko) 2021-07-01 2024-04-19 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 관련 방법
AU2022313258A1 (en) 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease
AU2022325231A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
CA3227108A1 (en) 2021-08-11 2023-02-16 Xiaomeng HU Genetically modified primary cells for allogeneic cell therapy
AU2022325955A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
JP2024531138A (ja) 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
AU2022326565A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
US20230090654A1 (en) 2021-08-24 2023-03-23 Homology Medicines, Inc. Adeno-associated virus formulations
WO2023028567A2 (en) * 2021-08-25 2023-03-02 Canbridge Pharmaceuticals, Inc. Aav particles comprising a liver-tropic capsid protein and acid alpha-glucosidase (gaa) and their use to treat pompe disease
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
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
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
CN118201947B (zh) * 2021-10-28 2025-03-21 上海天泽云泰生物医药有限公司 腺相关病毒衣壳
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023077085A2 (en) 2021-10-29 2023-05-04 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3238867A1 (en) 2021-11-29 2023-06-01 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
EP4440696A1 (en) 2021-12-01 2024-10-09 Shape Therapeutics Inc. Functional aav capsids for systemic administration
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
US20250099619A1 (en) * 2021-12-06 2025-03-27 Juntendo Educational Foundation Modified adeno-associated virus vector
US20230323395A1 (en) 2021-12-15 2023-10-12 Homology Medicines, Inc. Methods and compositions for the production of adeno-associated virus
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
US20250082777A1 (en) 2022-01-10 2025-03-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
AR128422A1 (es) 2022-02-02 2024-05-08 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
WO2023158836A1 (en) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
CA3245327A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. MODIFIED BATCH-BASED AAV PRODUCTION SYSTEMS AND PROCESSES
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
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
EP4504950A1 (en) 2022-04-01 2025-02-12 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
CA3247109A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
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
AU2023250660A1 (en) 2022-04-06 2024-10-24 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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
TW202417633A (zh) 2022-05-03 2024-05-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗TNF-α抑制劑
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
US20250375524A1 (en) 2022-06-30 2025-12-11 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
US20260027145A1 (en) 2022-07-27 2026-01-29 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
KR20250052387A (ko) 2022-08-25 2025-04-18 다케다 야쿠힌 고교 가부시키가이샤 파브리병의 치료에서의 사용을 위한 조성물
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding 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
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
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
CN116789739B (zh) * 2022-12-08 2024-01-26 广州派真生物技术有限公司 腺相关病毒突变体及其应用
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
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
WO2024146935A1 (en) 2023-01-06 2024-07-11 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
AU2023427408A1 (en) 2023-02-02 2025-09-04 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
JP2026513932A (ja) 2023-04-07 2026-05-01 リジェネクスバイオ インコーポレイテッド 組換えａａｖ生成のための組成物及び方法
EP4694904A2 (en) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting aav capsids, methods of manufacturing and using same
CN121311595A (zh) 2023-04-17 2026-01-09 阿斯克生物公司 转基因在灵长类动物中可调节的受控表达
CN121843705A (zh) 2023-05-03 2026-04-10 萨那生物科技公司 工程化胰岛细胞的给药和施用方法
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
EP4709866A2 (en) 2023-05-07 2026-03-18 REGENXBIO Inc. Compositions and methods for recombinant aav production
KR20260010739A (ko) 2023-05-11 2026-01-21 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 항불안 치료
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
JP2026522354A (ja) 2023-06-12 2026-07-07 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ｉｉｉｂのためのａａｖ遺伝子療法
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
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
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
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
KR20260114566A (ko) 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도
AU2024386366A1 (en) 2023-11-23 2026-06-04 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
TW202548022A (zh) 2024-03-03 2025-12-16 美商帕西奇生物公司 用於治療神經退化性病症之重組腺相關病毒
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses 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
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
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
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026036118A1 (en) 2024-08-09 2026-02-12 Shape Therapeutics Inc. Engineered aav capsid polypeptides and methods of use
WO2026044087A1 (en) 2024-08-21 2026-02-26 Shape Therapeutics Inc. Increased cellular stability for aav production
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
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
WO2026073032A1 (en) 2024-09-27 2026-04-02 Shape Therapeutics Inc. Constructs for improved aav production
WO2026094009A1 (en) 2024-11-04 2026-05-07 Insmed Incorporated Compositions and methods for treating duchenne muscular dystrophy
WO2026102076A1 (en) 2024-11-07 2026-05-15 Juno Therapeutics, Inc. Aav production method
WO2026114267A1 (en) 2024-11-29 2026-06-04 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating alzheimer's disease in primates
WO2026122921A1 (en) 2024-12-05 2026-06-11 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
EP4763209A1 (en) 2024-12-17 2026-06-24 Institut National de la Santé et de la Recherche Médicale Use of (alpha)s-setmar agonist for preventing or treating glioblastoma
WO2026137014A1 (en) 2024-12-20 2026-06-25 Samsung Bioepis Co., Ltd. Methods of purifying recombinant adeno-associated viruses
WO2026151888A1 (en) 2025-01-08 2026-07-16 The Trustees Of The University Of Pennsylvania Compositions for treating friedreich's ataxia
WO2026150302A1 (en) 2025-01-09 2026-07-16 Novartis Ag Novel aav rep/cap encoding constructs

Family Cites Families (76)

* 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
JPH07106156B2 (ja) 1983-10-28 1995-11-15 ジェネティックス、インスティチュ−ト ファクタ−▲ｖｉｉｉ▼および関連生産物の製造
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
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
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
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
DE69032043T2 (de) 1989-11-17 1998-06-04 Chiron Corp Proteinkomplexe mit faktor viii:c-aktivität und deren herstellung
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
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
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
WO1994011013A1 (en) 1992-11-13 1994-05-26 Duke University Chimeric blood coagulation proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
DK0874057T3 (da) 1993-06-10 2004-11-22 Bayer Ag Vektor og pattedyrcellelinie med forbedret produktionsevne
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
EP1696036B1 (en) * 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
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
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
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
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
CA2373352A1 (en) * 1999-07-02 2001-01-11 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
EP1228234A2 (en) * 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
DE60129799T2 (de) * 2000-03-31 2008-04-30 The General Hospital Corp., Boston Verfahren zur steigerung des haarwuchses durch wnt polypeptid
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
CA2453405A1 (en) * 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated viruses and uses thereof
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
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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
AU2003274397A1 (en) * 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20040002158A1 (en) * 2002-06-05 2004-01-01 Lung-Ji Chang Long term expression of lentiviral vectors
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
EP1545887B1 (en) 2002-09-30 2015-12-23 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
PL2657248T3 (pl) * 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
US8283151B2 (en) * 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
JP2009535339A (ja) * 2006-04-28 2009-10-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
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
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9434928B2 (en) * 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
JP6342886B2 (ja) * 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
NZ716102A (en) * 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
HUE052676T2 (hu) * 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
EP4378487A3 (en) 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
EP4134375A1 (en) * 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof
WO2020032986A1 (en) * 2018-08-10 2020-02-13 Logicbio Therapeutics, Inc. Non-disruptive gene therapy for the treatment of mma
US12630842B2 (en) * 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
JP2024534894A (ja) * 2021-09-03 2024-09-26 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法

Also Published As

Publication number Publication date
US11680274B2 (en) 2023-06-20
CN112029737A (zh) 2020-12-04
EP2383346A1 (en) 2011-11-02
US20210040504A1 (en) 2021-02-11
US20200208176A1 (en) 2020-07-02
EP3409296A1 (en) 2018-12-05
WO2006110689A3 (en) 2007-04-05
EP3085389A1 (en) 2016-10-26
EP1866422B1 (en) 2016-04-06
US10301648B2 (en) 2019-05-28
JP6039632B2 (ja) 2016-12-07
CN102994549A (zh) 2013-03-27
EP2359866A1 (en) 2011-08-24
DE23193601T1 (de) 2024-02-15
EP3603676A1 (en) 2020-02-05
JP2012165744A (ja) 2012-09-06
US20190218574A1 (en) 2019-07-18
JP2008538286A (ja) 2008-10-23
EP2359867B1 (en) 2014-10-08
EP2359865B1 (en) 2013-10-02
JP2015096070A (ja) 2015-05-21
WO2006110689A2 (en) 2006-10-19
CN101203613B (zh) 2012-12-12
EP1866422A2 (en) 2007-12-19
DK2359867T3 (en) 2015-01-05
EP2383346B1 (en) 2014-10-08
JP5702519B2 (ja) 2015-04-15
CN104293835A (zh) 2015-01-21
DK2383346T3 (en) 2015-01-05
US20150159173A1 (en) 2015-06-11
ES2580044T3 (es) 2016-08-18
EP4282956A3 (en) 2024-03-13
CN102994549B (zh) 2015-02-11
US20230272424A1 (en) 2023-08-31
US20170191079A1 (en) 2017-07-06
EP2359866B1 (en) 2013-07-17
US20090197338A1 (en) 2009-08-06
US10947561B2 (en) 2021-03-16
US20180057841A1 (en) 2018-03-01
US20240117378A1 (en) 2024-04-11
US10626415B2 (en) 2020-04-21
EP2359867A1 (en) 2011-08-24
CN101203613A (zh) 2008-06-18
ES2426245T3 (es) 2013-10-22
US20230416780A1 (en) 2023-12-28
US20230287452A1 (en) 2023-09-14
JP2014204723A (ja) 2014-10-30
EP4282957A3 (en) 2024-03-13
ES2525067T3 (es) 2014-12-17
EP2357010B1 (en) 2013-06-12
JP2016168047A (ja) 2016-09-23
CN117363655A (zh) 2024-01-09
EP4282957A2 (en) 2023-11-29
JP6275769B2 (ja) 2018-02-07
US20250171801A1 (en) 2025-05-29
JP5677681B2 (ja) 2015-02-25
US8999678B2 (en) 2015-04-07
EP2357010A1 (en) 2011-08-17
CN106906241B (zh) 2020-09-11
CN104293835B (zh) 2017-07-04
ES2428218T3 (es) 2013-11-06
EP2359865A1 (en) 2011-08-24
EP4234687A2 (en) 2023-08-30
EP4282956A2 (en) 2023-11-29
JP5912147B2 (ja) 2016-04-27
ES2525143T3 (es) 2014-12-18
CN106906241A (zh) 2017-06-30
US20250179525A1 (en) 2025-06-05
EP3603676A8 (en) 2020-03-25
US20230340532A1 (en) 2023-10-26
ES2434723T3 (es) 2013-12-17
US20200325491A1 (en) 2020-10-15

Similar Documents

Publication Publication Date Title
DE23193601T1 (de) Verfahren zur erhöhung der funktion eines aav-vektors
US8105831B2 (en) Parvoviral production of HLA homozygous cells
MX2023012410A (es) Metodos para tratar enfermedades oculares usando variantes de aav2 que codifican aflibercept.
CA2407309A1 (en) Dna sequences encoding dystrophin minigenes and methods of use thereof
ATE445006T1 (de) Differenzierung von nicht-insulin in insulin- produzierende zellen durch glp-1 und exendin-4 und dessen verwendung
US20230193315A1 (en) Methods for using transcription-dependent directed evolution of aav capsids
CA2349838A1 (en) Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
NZ600121A (en) A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
AU2025203194A1 (en) Nucleic acid molecules containing spacers and methods of use thereof
JP2025507958A (ja) 操作された核内低分子ＲＮＡ（ｓｎＲＮＡ）を含む組成物および方法
CN111218446A (zh) 一种肝脏特异性启动子及其应用
CN111235158A (zh) 用于表达重组人β-珠蛋白的病毒载体及其应用
JP2023515672A5 (enExample)
DE1066395T1 (de) Induzierendes alphavirengen- expressionssystem
EP4682163A3 (en) Method of reducing cst fluctuation in neovascular amd by a recombinant adeno-associated virus
CN102127546B (zh) 一种骨骼肌特异性actin启动子及其应用
Ishizaki et al. Brain secretory peptides of the silkmoth Bombyx mori: prothoracicotropic hormone and bombyxin
DE19644500C2 (de) AAV-DNA mit Helfervirus-Sequenzen
WO2022251096A1 (en) Promoter sequence and related products and uses thereof
DE19608751B4 (de) Verwendung eines Adeno-assoziierten Virus-Vektors zur Steigerung der Immunogenität von Zellen
DE69320154T2 (de) Menschliche Id-Gene
US20250304920A1 (en) Compositions and Methods for Enhanced Production of Adeno-Associated Virus
EP4570260A1 (en) Viral vector for transduction of adipocytes
DE19625188A1 (de) System zur Herstellung von AAV-Vektoren
RU2025130605A (ru) Варианты aav для лечения расстройств системы комплемента