KR102604510B1 - 신규 아데노-관련 바이러스 캡시드 단백질 - Google Patents

신규 아데노-관련 바이러스 캡시드 단백질 Download PDF

Info

Publication number
KR102604510B1
KR102604510B1 KR1020197001930A KR20197001930A KR102604510B1 KR 102604510 B1 KR102604510 B1 KR 102604510B1 KR 1020197001930 A KR1020197001930 A KR 1020197001930A KR 20197001930 A KR20197001930 A KR 20197001930A KR 102604510 B1 KR102604510 B1 KR 102604510B1
Authority
KR
South Korea
Prior art keywords
gly
asn
pro
thr
ala
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.)
Active
Application number
KR1020197001930A
Other languages
English (en)
Korean (ko)
Other versions
KR20190032375A (ko
Inventor
피터 카메론 콜로시
마이클 로취리
로버트 엔지
Original Assignee
바이오마린 파머수티컬 인크.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 바이오마린 파머수티컬 인크. filed Critical 바이오마린 파머수티컬 인크.
Priority to KR1020237039466A priority Critical patent/KR20230161535A/ko
Publication of KR20190032375A publication Critical patent/KR20190032375A/ko
Application granted granted Critical
Publication of KR102604510B1 publication Critical patent/KR102604510B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • C12N2750/14143Use 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14144Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Toxicology (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)
  • Saccharide Compounds (AREA)
KR1020197001930A 2016-07-26 2017-07-25 신규 아데노-관련 바이러스 캡시드 단백질 Active KR102604510B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020237039466A KR20230161535A (ko) 2016-07-26 2017-07-25 신규 아데노-관련 바이러스 캡시드 단백질

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662366838P 2016-07-26 2016-07-26
US62/366,838 2016-07-26
PCT/US2017/043703 WO2018022608A2 (en) 2016-07-26 2017-07-25 Novel adeno-associated virus capsid proteins

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020237039466A Division KR20230161535A (ko) 2016-07-26 2017-07-25 신규 아데노-관련 바이러스 캡시드 단백질

Publications (2)

Publication Number Publication Date
KR20190032375A KR20190032375A (ko) 2019-03-27
KR102604510B1 true KR102604510B1 (ko) 2023-11-23

Family

ID=59558468

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020237039466A Abandoned KR20230161535A (ko) 2016-07-26 2017-07-25 신규 아데노-관련 바이러스 캡시드 단백질
KR1020197001930A Active KR102604510B1 (ko) 2016-07-26 2017-07-25 신규 아데노-관련 바이러스 캡시드 단백질

Family Applications Before (1)

Application Number Title Priority Date Filing Date
KR1020237039466A Abandoned KR20230161535A (ko) 2016-07-26 2017-07-25 신규 아데노-관련 바이러스 캡시드 단백질

Country Status (15)

Country Link
US (3) US11584780B2 (enExample)
EP (1) EP3491008A2 (enExample)
JP (4) JP6994018B2 (enExample)
KR (2) KR20230161535A (enExample)
CN (1) CN109715650B (enExample)
AU (2) AU2017301600B2 (enExample)
BR (1) BR112019001532A2 (enExample)
CA (1) CA3031026A1 (enExample)
CL (6) CL2019000167A1 (enExample)
IL (2) IL305149A (enExample)
MX (1) MX2019000962A (enExample)
PE (1) PE20190401A1 (enExample)
PH (1) PH12019500068A1 (enExample)
SG (1) SG11201811603WA (enExample)
WO (1) WO2018022608A2 (enExample)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP4219724A3 (en) 2016-08-23 2023-09-27 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12416016B2 (en) * 2018-02-27 2025-09-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ,インコーポレイテッド 抗体を回避するウイルスベクター
MA51353B1 (fr) * 2018-04-05 2022-09-30 Univ Sorbonne Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
BR112020022858A2 (pt) * 2018-05-11 2021-02-23 Massachusetts Eye And Ear Infirmary tropismo específico hepático de vírus adeno-associado
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
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
JP7451497B2 (ja) * 2018-08-21 2024-03-18 マサチューセッツ アイ アンド イヤー インファーマリー アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法
WO2020117898A1 (en) * 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
US20220133909A1 (en) * 2019-01-23 2022-05-05 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
WO2020198737A1 (en) * 2019-03-28 2020-10-01 The General Hospital Corporation Engineered adeno-associated (aav) vectors for transgene expression
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
US20220403414A1 (en) * 2019-10-16 2022-12-22 Wuxi Apptec (Shanghai) Co., Ltd. Novel aav variant
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CA3161154A1 (en) 2019-11-14 2021-05-20 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with liver-specific gene therapy vectors
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
CN115836081A (zh) * 2020-02-25 2023-03-21 儿童医学研究院 腺相关病毒衣壳多肽及载体
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
IL297753A (en) 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
EP4176064A1 (en) * 2020-07-03 2023-05-10 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
US20240209030A1 (en) * 2020-09-10 2024-06-27 Genethon Peptide-Modified AAV Capsid
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
CA3195553A1 (en) * 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
MX2023005041A (es) 2020-11-02 2023-05-17 Biomarin Pharm Inc Proceso para enriquecer virus adenoasociado.
US20240376495A1 (en) 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034996A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034989A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
AR127217A1 (es) 2021-10-01 2023-12-27 Biomarin Pharm Inc Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
US20250213727A1 (en) * 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
IL319714A (en) 2022-09-22 2025-05-01 Dinaqor Ag Treating cardiac myopathy with AAV gene therapy vectors
TW202421788A (zh) 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用aav基因療法載體治療致心律不整性心肌病
CN115925819B (zh) * 2022-12-30 2023-10-13 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2025051970A2 (en) 2023-09-06 2025-03-13 Dinaqor Ag Methods of treatment of hypertrophic cardiomyopathy with aav gene therapy vectors and therapeutic formulations
WO2025054465A1 (en) * 2023-09-08 2025-03-13 California Institute Of Technology Compositions for transport of therapeutic cargos using miniprotein binders targeting ca-iv
WO2025104321A1 (en) * 2023-11-17 2025-05-22 Genethon Prediction of multiple aav properties by deep learning
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (70)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
DK518384A (da) 1984-01-31 1985-07-01 Idaho Res Found Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse
US4994371A (en) 1987-08-28 1991-02-19 Davie Earl W DNA preparation of Christmas factor and use of DNA sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6309853B1 (en) 1994-08-17 2001-10-30 The Rockfeller University Modulators of body weight, corresponding nucleic acids and proteins, and diagnostic and therapeutic uses thereof
RU2273645C9 (ru) 1994-08-17 2006-11-27 Дзе Рокефеллер Юниверсити Полипептид ожирения (ов)(варианты), его аналог (варианты) и слитый белок (варианты), изолированная молекула нуклеиновой кислоты, молекула днк, рекомбинантный вектор клонирования, рекомбинантный вектор экспрессии, фармацевтическая композиция, моноклональное и поликлональное антитело
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1999003496A1 (en) 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
JP4169478B2 (ja) 1998-04-21 2008-10-22 マイクロメット アーゲー Cd19×cd3特異的ポリペプチドおよびその使用
JP2004514407A (ja) 2000-04-28 2004-05-20 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 異種キャプシド中にシュードタイピングされたaav5キャプシドおよびaav5ベクターを含む組換えaavベクター
CA2470711C (en) 2001-10-16 2011-07-12 National Institute Of Advanced Industrial Science And Technology Novel n-acetylglucosamine transferase, nucleic acid encoding the same, antibody against the same and use thereof for diagnosing cancer or tumor
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP2573170B1 (en) 2001-12-17 2017-12-20 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
AU2003237159A1 (en) 2002-04-30 2003-11-17 University Of Florida Treatment for phenylketonuria
ES2874298T3 (es) 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US7732599B2 (en) 2003-11-17 2010-06-08 Biomarin Pharmaceutical Inc. Process for preparing tetrahydrobiopterin and analogs of tetrahydrobiopterin
EP1771571A2 (en) 2004-07-30 2007-04-11 Targeted Genetics Corporation Recombinant aav based vaccine methods
CN101132776A (zh) 2004-11-17 2008-02-27 生物马林药物股份有限公司 稳定的四氢生物喋呤片剂制剂
ES2428218T3 (es) 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
US7534595B2 (en) 2006-06-12 2009-05-19 Biomarin Pharmaceutical Inc. Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof
US7531341B1 (en) 2006-06-12 2009-05-12 Biomarin Pharmaceutical Inc. Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
AR066354A1 (es) 2007-05-01 2009-08-12 Genzyme Corp La terapia sistematica con factor-1 de crecimiento de tipo insulina reduce la neuropatia periferica diabetica y mejora la funcion renal en la nefropatia diabetica
BRPI0814459B1 (pt) 2007-07-26 2023-01-24 Uniqure Ip B.V Método para produzir um vírion parvoviral recombinante em uma célula de inseto, e, construção de ácido nucleico
US7560263B2 (en) 2007-08-17 2009-07-14 Biomarin Pharmaceutical Inc. Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating cancer using compositions thereof
CN101959891A (zh) 2008-01-07 2011-01-26 生物马林药物股份有限公司 合成四氢生物蝶呤的方法
RU2502800C2 (ru) 2008-01-15 2013-12-27 Эббви Инк. Усовершенствованные векторы экспрессии млекопитающих и их применение
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
WO2010014225A2 (en) 2008-07-30 2010-02-04 Biomarin Pharmaceutical Inc. Assays for detection of phenylalanine ammonia-lyase and antibodies to phenylalanine ammonia-lyase
ES2985035T3 (es) 2008-09-15 2024-11-04 Uniqure Biopharma B V Mutante de polipéptido de factor IX, sus usos y su procedimiento de producción
DK2425000T3 (da) * 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
TWI688395B (zh) 2010-03-23 2020-03-21 英翠克頌公司 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
JP6373763B2 (ja) 2012-02-17 2018-08-22 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ベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
KR102346455B1 (ko) 2013-03-15 2022-01-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Mpsi 치료를 위한 조성물 및 방법
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
MX2016001044A (es) 2013-07-22 2016-09-09 The Children's Hospital Of Philadelphia Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
SI3044231T1 (sl) 2013-09-12 2020-12-31 Biomarin Pharmaceutical Inc. Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII
WO2015054653A2 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
DK3116900T3 (da) 2014-03-09 2020-09-28 Univ Pennsylvania Sammensætninger som kan anvendes til behandling af ornithintranscarbamylase (otc) defekt
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
AU2015283954B2 (en) 2014-07-02 2020-11-12 Translate Bio, Inc. Encapsulation of messenger RNA
US10301367B2 (en) 2014-07-26 2019-05-28 Consiglio Nazionale Delle Ricerche Compositions and methods for treatment of muscular dystrophy
PL3198018T3 (pl) 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
PT3872085T (pt) * 2015-07-30 2023-04-13 Massachusetts Eye & Ear Infirmary Sequências de aav ancestrais e utilizações destas
RU2020126034A (ru) 2015-09-24 2020-09-02 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
WO2017066764A2 (en) * 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
EP3368664A4 (en) 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
PL3534922T3 (pl) 2016-11-04 2024-08-19 Takeda Pharmaceutical Company Limited Preparaty wirusa związanego z adenowirusem
CN110325199A (zh) 2016-12-30 2019-10-11 宾夕法尼亚州立大学托管会 用于治疗苯丙酮尿症的基因疗法
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7066861B2 (ja) 2018-02-01 2022-05-13 ホモロジー・メディシンズ・インコーポレイテッド Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
KR20210008491A (ko) 2018-05-14 2021-01-22 바이오마린 파머수티컬 인크. 청소년 대상체에서 aav 벡터의 안정한 발현
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
EP3826719A1 (en) 2018-07-24 2021-06-02 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
MX2021012564A (es) 2019-04-19 2022-01-18 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados.
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Proc. Natl. Acad. Sci. USA, Vol.97, No.25, pp.13714-13719(2000.12.05.)*

Also Published As

Publication number Publication date
CN109715650B (zh) 2024-03-08
JP7303287B2 (ja) 2023-07-04
CA3031026A1 (en) 2018-02-01
JP2022001064A (ja) 2022-01-06
IL263801B1 (en) 2023-09-01
MX2019000962A (es) 2019-08-01
BR112019001532A2 (pt) 2019-09-10
AU2017301600A1 (en) 2019-01-17
NZ749467A (en) 2024-08-30
JP2019525758A (ja) 2019-09-12
WO2018022608A3 (en) 2018-03-01
JP2023076606A (ja) 2023-06-01
CL2023000165A1 (es) 2023-09-08
JP7395550B2 (ja) 2023-12-11
PE20190401A1 (es) 2019-03-13
KR20230161535A (ko) 2023-11-27
US20230322864A1 (en) 2023-10-12
EP3491008A2 (en) 2019-06-05
CL2023003010A1 (es) 2024-04-26
JP6994018B2 (ja) 2022-01-14
SG11201811603WA (en) 2019-02-27
CL2023002764A1 (es) 2024-04-05
RU2019103983A (ru) 2020-08-26
AU2017301600B2 (en) 2022-08-04
CN109715650A (zh) 2019-05-03
PH12019500068A1 (en) 2019-10-21
KR20190032375A (ko) 2019-03-27
CL2019000167A1 (es) 2019-05-24
IL305149A (en) 2023-10-01
AU2022259813A1 (en) 2022-12-01
US20240317813A1 (en) 2024-09-26
CL2020003176A1 (es) 2021-06-04
JP2022033245A (ja) 2022-02-28
RU2019103983A3 (enExample) 2021-03-03
WO2018022608A2 (en) 2018-02-01
CL2023003001A1 (es) 2024-04-19
IL263801B2 (en) 2024-01-01
US20220402974A1 (en) 2022-12-22
US11584780B2 (en) 2023-02-21
IL263801A (en) 2019-01-31

Similar Documents

Publication Publication Date Title
JP7303287B2 (ja) 新規アデノ随伴ウイルスキャプシドタンパク質
US11821008B2 (en) Liver targeting adeno-associated viral vectors
US20240200096A1 (en) Methods of redosing gene therapy vectors
CA2406743A1 (en) Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20240132910A1 (en) USE OF HISTIDINE RICH PEPTIDES AS A TRANSFECTION REAGENT FOR rAAV AND rBV PRODUCTION
RU2793735C2 (ru) Новые нацеливающие на печень аденоассоциированные вирусные векторы
RU2779318C2 (ru) Новые капсидные белки адено-ассоциированного вируса

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

AMND Amendment
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

AMND Amendment
P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

AMND Amendment
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E601 Decision to refuse application
PE0601 Decision on rejection of patent

St.27 status event code: N-2-6-B10-B15-exm-PE0601

AMND Amendment
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PX0901 Re-examination

St.27 status event code: A-2-3-E10-E12-rex-PX0901

PX0701 Decision of registration after re-examination

St.27 status event code: A-3-4-F10-F13-rex-PX0701

X701 Decision to grant (after re-examination)
PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A17-div-PA0104

St.27 status event code: A-0-1-A10-A16-div-PA0104

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601