JP6767483B2 - 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター - Google Patents

遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター Download PDF

Info

Publication number
JP6767483B2
JP6767483B2 JP2018521395A JP2018521395A JP6767483B2 JP 6767483 B2 JP6767483 B2 JP 6767483B2 JP 2018521395 A JP2018521395 A JP 2018521395A JP 2018521395 A JP2018521395 A JP 2018521395A JP 6767483 B2 JP6767483 B2 JP 6767483B2
Authority
JP
Japan
Prior art keywords
nucleic acid
fxn
seq
sequence
vector
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.)
Expired - Fee Related
Application number
JP2018521395A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018531609A (ja
JP2018531609A5 (enExample
Inventor
ジェイ. サムルスキー リチャード
ジェイ. サムルスキー リチャード
Original Assignee
バンブー セラペウティクス インク
バンブー セラペウティクス インク
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 バンブー セラペウティクス インク, バンブー セラペウティクス インク filed Critical バンブー セラペウティクス インク
Publication of JP2018531609A publication Critical patent/JP2018531609A/ja
Publication of JP2018531609A5 publication Critical patent/JP2018531609A5/ja
Application granted granted Critical
Publication of JP6767483B2 publication Critical patent/JP6767483B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Physics & Mathematics (AREA)
JP2018521395A 2015-11-05 2016-11-01 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター Expired - Fee Related JP6767483B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562251288P 2015-11-05 2015-11-05
US62/251,288 2015-11-05
US201662411980P 2016-10-24 2016-10-24
US62/411,980 2016-10-24
PCT/IB2016/056572 WO2017077451A1 (en) 2015-11-05 2016-11-01 Modified friedreich ataxia genes and vectors for gene therapy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020156320A Division JP2021019591A (ja) 2015-11-05 2020-09-17 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター

Publications (3)

Publication Number Publication Date
JP2018531609A JP2018531609A (ja) 2018-11-01
JP2018531609A5 JP2018531609A5 (enExample) 2019-12-12
JP6767483B2 true JP6767483B2 (ja) 2020-10-14

Family

ID=57288476

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018521395A Expired - Fee Related JP6767483B2 (ja) 2015-11-05 2016-11-01 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター
JP2020156320A Pending JP2021019591A (ja) 2015-11-05 2020-09-17 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2020156320A Pending JP2021019591A (ja) 2015-11-05 2020-09-17 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター

Country Status (18)

Country Link
US (2) US10548947B2 (enExample)
EP (1) EP3370778A1 (enExample)
JP (2) JP6767483B2 (enExample)
KR (2) KR20210005994A (enExample)
CN (1) CN108348621A (enExample)
AU (2) AU2016347887B2 (enExample)
BR (1) BR112018007453A2 (enExample)
CA (1) CA2947584A1 (enExample)
CO (1) CO2018004564A2 (enExample)
HK (1) HK1250924A1 (enExample)
IL (1) IL259141A (enExample)
MX (1) MX2018005084A (enExample)
PE (1) PE20181156A1 (enExample)
PH (1) PH12018500964A1 (enExample)
RU (2) RU2021102893A (enExample)
SG (2) SG10201912763QA (enExample)
TW (2) TWI654301B (enExample)
WO (1) WO2017077451A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021019591A (ja) * 2015-11-05 2021-02-18 バンブー セラペウティクス インク 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター

Families Citing this family (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10617770B2 (en) * 2015-04-24 2020-04-14 University Of Florida Research Foundation, Incorporated AAV vector for treatment of Friedreich's ataxia
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
RU2726131C2 (ru) 2015-05-22 2020-07-09 Дмитрий Дмитриевич Генкин Внеклеточная днк в качестве терапевтической мишени при нейродегенерации
US12011488B2 (en) 2016-03-23 2024-06-18 The Regents Of The University Of California Methods of treating mitochondrial disorders
US12012436B2 (en) * 2016-03-23 2024-06-18 The Regents Of The University Of California Methods of treating mitochondrial disorders
US10935747B2 (en) 2016-09-21 2021-03-02 Commscope Technologies Llc Adapter block assembly
US11116852B2 (en) 2016-11-09 2021-09-14 Precigen, Inc. Frataxin expression constructs
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.
CN112553210B (zh) 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020006633A2 (pt) 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
EP3697914B9 (en) * 2017-10-17 2023-10-04 Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol Vectors for the treatment of friedreich's ataxia
US11905522B2 (en) 2018-01-16 2024-02-20 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス 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
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US20210308274A1 (en) * 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
JP7473548B2 (ja) * 2018-08-10 2024-04-23 ロジックバイオ セラピューティクス, インコーポレイテッド Mmaの処置のための非破壊的遺伝子治療
CN113383010B (zh) * 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
CA3120082A1 (en) * 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
AU2019381776A1 (en) * 2018-11-16 2021-07-01 AskBio Inc. Therapeutic adeno-associated virus for treating Pompe disease
AU2019385506A1 (en) * 2018-11-21 2021-06-03 Takeda Pharmaceutical Company Limited Recombinant viral vectors and nucleic acids for producing the same
WO2020132647A1 (en) * 2018-12-21 2020-06-25 Northwestern University Use of annexins in preventing and treating muscle membrane injury
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
US20230123802A1 (en) * 2019-03-27 2023-04-20 Sigilon Therapeutics, Inc. Compositions, devices, and methods for factor vii therapy
CN113993994A (zh) * 2019-03-28 2022-01-28 因特利亚治疗公司 用于多肽表达的多核苷酸、组合物和方法
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
US20230144548A1 (en) * 2019-06-30 2023-05-11 John Fraser Wright Recombinant aav vectors with altered immunogencity and methods of making the same
WO2021000024A1 (en) * 2019-07-04 2021-01-07 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
US20220241394A1 (en) * 2019-07-12 2022-08-04 Cls Therapeutics Limited TREATMENT OF DISEASES ASSOCIATED WITH PROTEIN MISFOLDING BY NERVOUS SYSTEM EXPRESSION OF AN ENZYME WHICH HAS A DEOXYRIBONUCLEASE (DNase) ACTIVITY
AU2020346914A1 (en) * 2019-09-13 2022-04-21 Prevail Therapeutics, Inc. Compositions and methods for treatment of Friedreich's Ataxia
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
IL293972A (en) * 2019-12-19 2022-08-01 Univ Pennsylvania Preparations for the treatment of Friedrich's ataxia
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神(北京)生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
WO2022026516A1 (en) * 2020-07-29 2022-02-03 The Board Of Regents Of The University Of Texas System Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
IL302608A (en) 2020-11-03 2023-07-01 Pfizer Methods for purification of aav vectors by anion exchange chromatography
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of aav vectors by affinity chromatography
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
US20240050521A1 (en) * 2021-01-11 2024-02-15 The Trustees Of The University Of Pennsylvania Compositions for treating friedreich's ataxia
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024011109A2 (en) * 2022-07-06 2024-01-11 Adverum Biotechnologies, Inc. Compositions and methods for treatment of achromotopsia
CN116121350A (zh) * 2022-12-02 2023-05-16 百码科技(深圳)有限公司 一种检测fxn致病基因突变的方法
CN117089514A (zh) * 2023-10-13 2023-11-21 思鹏生物科技(苏州)有限公司 提升hek293细胞系aav生产效率的细胞筛选驯化方法
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2221293C (en) 1995-06-07 2012-09-18 University Of North Carolina At Chapel Hill Helper virus-free aav production
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV5 vector and uses thereof
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
AU2002240890A1 (en) 2000-12-07 2002-06-18 Universite De Nantes Inducible highly productive raav packaging cell-lines
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
EP2185591B1 (en) * 2007-08-20 2014-05-14 Glaxo Group Limited Production method
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
AU2010340358B2 (en) * 2009-12-21 2014-07-24 Pharmathene, Inc. Recombinant butyrylcholinesterases and truncates thereof
AU2011213559B2 (en) * 2010-02-08 2015-05-07 The Trustees Of The University Of Pennsylvania Nucleic acid molecules encoding RANTES, and compositions comprising and methods of using the same
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
RU2620980C2 (ru) * 2011-06-09 2017-05-30 Курна, Инк. Лечение заболеваний, связанных с фратаксином (fxn), путем ингибирования природного антисмыслового транскрипта fxn
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
US9066966B2 (en) * 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
AU2014229051B2 (en) 2013-03-15 2020-07-16 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
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
AU2014287005C1 (en) 2013-07-12 2021-06-03 The Children's Hospital Of Philadelphia AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies
PT3024498T (pt) 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
PE20181156A1 (es) * 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021019591A (ja) * 2015-11-05 2021-02-18 バンブー セラペウティクス インク 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター

Also Published As

Publication number Publication date
TWI743442B (zh) 2021-10-21
SG10201912763QA (en) 2020-02-27
US20170128528A1 (en) 2017-05-11
SG11201802408RA (en) 2018-05-30
AU2020200976A1 (en) 2020-02-27
TW201920673A (zh) 2019-06-01
TW201723179A (zh) 2017-07-01
CA2947584A1 (en) 2017-05-05
TWI654301B (zh) 2019-03-21
RU2743792C2 (ru) 2021-02-26
AU2016347887A1 (en) 2018-04-19
CO2018004564A2 (es) 2018-12-14
KR20180077275A (ko) 2018-07-06
AU2020200976B2 (en) 2021-09-30
KR102200642B1 (ko) 2021-01-12
RU2018116076A3 (enExample) 2020-08-21
PE20181156A1 (es) 2018-07-19
BR112018007453A2 (pt) 2018-10-23
JP2018531609A (ja) 2018-11-01
RU2021102893A (ru) 2021-03-03
HK1250924A1 (zh) 2019-01-18
JP2021019591A (ja) 2021-02-18
IL259141A (en) 2018-06-28
RU2018116076A (ru) 2019-10-28
MX2018005084A (es) 2019-05-16
US10548947B2 (en) 2020-02-04
WO2017077451A1 (en) 2017-05-11
CN108348621A (zh) 2018-07-31
EP3370778A1 (en) 2018-09-12
PH12018500964A1 (en) 2018-11-12
AU2016347887B2 (en) 2019-11-14
US20200384073A1 (en) 2020-12-10
KR20210005994A (ko) 2021-01-15

Similar Documents

Publication Publication Date Title
JP2021019591A (ja) 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター
KR102526616B1 (ko) 윌슨병 및 기타 병태의 치료에 사용되기 위한 핵산 구조물 및 유전자 치료용 벡터
JP2021045136A (ja) 最適化されたミニ−ジストロフィン遺伝子および発現カセットおよびそれらの使用
JP2021121638A (ja) 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
KR20180091863A (ko) 임상적 사용에 적합한 무혈청 현탁 세포 배양 시스템에서 재조합 아데노-관련 바이러스(aav) 벡터를 생성하기 위한 확장가능한 방법
KR20160026841A (ko) 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법
WO2015124546A1 (en) Aav vectors for the treatment of ischemic and non-ischemic heart disease
US20240091383A1 (en) Synergistic effect of smn1 and mir-23a in treating spinal muscular atrophy
EP4159863A1 (en) Codon-optimized nucleic acid encoding smn1 protein
US20190290781A1 (en) Methods and Compositions for Treating Dystroglycanopathy Disorders
US20220389456A1 (en) Modified soluble vegf receptor-1 genes and vectors for gene therapy
CN115772520B (zh) 用于治疗庞贝氏病的基因治疗构建体、药物组合物和方法
CN112011571A (zh) 一种用于治疗脊髓性肌萎缩的基因治疗药物
US20230338582A1 (en) Methods of Treating Human X-Linked Retinoschisis Using Gene Therapy
WO2023131345A1 (zh) 用于x染色体连锁肾上腺脑白质营养不良的基因治疗药物和方法
CN118696126A (zh) 用于治疗雷特综合征及相关病症的重组优化的mecp2盒和方法
CN116179605B (zh) 一种重组腺相关病毒载体及其应用
RU2825667C2 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе
EP4198134A1 (en) Gamma-sarcoglycan gene transfer increase using modified itr sequences
WO2025217117A1 (en) Compositions with cardiac and skeletal muscle-specific targeting motifs and uses thereof
WO2023111102A1 (en) Alpha-sarcoglycan gene transfer increase using modified itr sequences
HK40116356A (zh) 用於治疗雷特综合征及相关病症的重组优化的mecp2盒和方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191030

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20191030

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20191108

A975 Report on accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A971005

Effective date: 20191121

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20191210

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200225

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200402

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200528

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20200825

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20200917

R150 Certificate of patent or registration of utility model

Ref document number: 6767483

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees