PT2527457T - Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav - Google Patents

Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav

Info

Publication number
PT2527457T
PT2527457T PT12180951T PT12180951T PT2527457T PT 2527457 T PT2527457 T PT 2527457T PT 12180951 T PT12180951 T PT 12180951T PT 12180951 T PT12180951 T PT 12180951T PT 2527457 T PT2527457 T PT 2527457T
Authority
PT
Portugal
Prior art keywords
gene delivery
motor neurons
aav vectors
peripheral injection
widespread gene
Prior art date
Application number
PT12180951T
Other languages
English (en)
Inventor
Barkats Martine
Original Assignee
Genethon
Centre Nat Rech Scient
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=39186802&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PT2527457(T) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genethon, Centre Nat Rech Scient filed Critical Genethon
Publication of PT2527457T publication Critical patent/PT2527457T/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • 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
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • A61K48/0083Medicinal 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 administration regime
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pain & Pain Management (AREA)
  • Transplantation (AREA)
  • Hospice & Palliative Care (AREA)
  • Anesthesiology (AREA)
  • Rheumatology (AREA)
  • Psychiatry (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
PT12180951T 2007-10-05 2008-10-03 Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav PT2527457T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP07301435A EP2058401A1 (en) 2007-10-05 2007-10-05 Widespread gene delivery to motor neurons using peripheral injection of AAV vectors

Publications (1)

Publication Number Publication Date
PT2527457T true PT2527457T (pt) 2019-05-31

Family

ID=39186802

Family Applications (3)

Application Number Title Priority Date Filing Date
PT88367768T PT2212424E (pt) 2007-10-05 2008-10-03 Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav
PT12180951T PT2527457T (pt) 2007-10-05 2008-10-03 Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav
PT191581529T PT3505635T (pt) 2007-10-05 2008-10-03 Entrega generalizada de genes nos neurónios motores por injeção periférica de vetores aav

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT88367768T PT2212424E (pt) 2007-10-05 2008-10-03 Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav

Family Applications After (1)

Application Number Title Priority Date Filing Date
PT191581529T PT3505635T (pt) 2007-10-05 2008-10-03 Entrega generalizada de genes nos neurónios motores por injeção periférica de vetores aav

Country Status (18)

Country Link
US (3) US9926574B2 (pt)
EP (5) EP2058401A1 (pt)
JP (2) JP5620819B2 (pt)
CN (2) CN101883859A (pt)
CA (2) CA2999607A1 (pt)
CY (1) CY1125608T1 (pt)
DK (2) DK2212424T3 (pt)
ES (3) ES2924892T3 (pt)
FI (1) FI2212424T4 (pt)
FR (2) FR20C1057I2 (pt)
HR (2) HRP20220992T3 (pt)
HU (2) HUE059864T2 (pt)
LT (2) LT3505635T (pt)
NO (1) NO2022048I1 (pt)
PL (2) PL3505635T3 (pt)
PT (3) PT2212424E (pt)
SI (2) SI3505635T1 (pt)
WO (1) WO2009043936A1 (pt)

Families Citing this family (95)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
ES2796085T3 (es) 2008-01-15 2020-11-25 Univ Leland Stanford Junior Marcadores de células madre de leucemia mieloide aguda
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
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
MX356669B (es) 2009-05-02 2018-06-08 Genzyme Corp Terapia genica para trastornos neurodegenerativos.
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
JP2013533847A (ja) 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ コレステロール関連障害のaavベースの治療
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
CA3049237C (en) * 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CN103189507A (zh) * 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
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
JP2015523998A (ja) 2012-06-19 2015-08-20 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 疾患を治療するための組成物および方法
CA2877428A1 (en) 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
JP6314138B2 (ja) 2012-08-01 2018-04-18 ネイションワイド チルドレンズ ホスピタル 組換えアデノ随伴ウイルス9の髄腔内送達
CA2885216A1 (en) 2012-09-17 2014-03-20 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
KR20240093939A (ko) * 2013-05-15 2024-06-24 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
ES2936220T3 (es) 2013-08-27 2023-03-15 Res Inst Nationwide Childrens Hospital Productos y métodos para el tratamiento de la esclerosis lateral amiotrófica
JP2016538276A (ja) 2013-11-05 2016-12-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルThe Research Institute At Nationwide Children’S Hospital 筋萎縮性側索硬化症の処置のためのNF−κBおよびSOD−1を阻害する組成物および方法
KR102390075B1 (ko) 2014-03-09 2022-04-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 오르니틴 트랜스카르바밀라아제(otc) 결핍증의 치료에 유용한 조성물
EP2933335A1 (en) * 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2016016449A1 (en) 2014-07-31 2016-02-04 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるaavベクター
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
KR102599909B1 (ko) 2014-11-14 2023-11-09 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
EP4344741A2 (en) 2014-11-21 2024-04-03 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
AU2015364636B9 (en) 2014-12-16 2021-12-02 Board Of Regents Of The University Of Nebraska Gene therapy for Juvenile Batten Disease
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
ES2755725T3 (es) * 2015-04-08 2020-04-23 Us Health Terapia génica viral como tratamiento para una enfermedad o un trastorno por almacenamiento de colesterol
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
BR112018006480A2 (pt) * 2015-10-01 2018-10-09 Goleini Inc ?vetor, método de tratamento de uma doença ou condição, composição, combinação de um vetor, kit, uso de um vetor, método para a modulação in vivo da atividade eletrofisiológica de uma célula
EP3365438B1 (en) * 2015-10-21 2021-04-28 The United States of America, as represented by The Secretary, Department of Health and Human Services Codon-optimized reduced-size atp7a cdna and uses for treatment of copper transport disorders
CA3002982A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
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
AU2017321488B2 (en) 2016-08-30 2022-10-20 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
CN108524952A (zh) * 2017-03-06 2018-09-14 复旦大学 腺相关病毒aav9及其在制备治疗戈谢氏病的制剂中的用途
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TWI804518B (zh) 2017-10-16 2023-06-11 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
US20210317474A1 (en) * 2017-11-08 2021-10-14 Novartis Ag Means and method for producing and purifying viral vectors
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019161365A1 (en) 2018-02-19 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
MA52631A (fr) 2018-05-15 2021-03-24 Voyager Therapeutics Inc Compositions et méthodes pour le traitement de la maladie de parkinson
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
US20210361318A1 (en) 2018-07-02 2021-11-25 Voyager Therapeutics, Inc. Cannula system and use thereof
WO2020010042A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
CA3107462A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2020208467A1 (en) 2019-01-18 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing AAV particles
KR20210125014A (ko) * 2019-02-05 2021-10-15 더 트러스티즈 오브 프린스턴 유니버시티 알파헤르페스바이러스 프로모터 서열을 함유하는 재조합 핵산
CN109706176A (zh) * 2019-03-12 2019-05-03 青岛大学附属医院 可靶向转染背根神经节卫星胶质细胞腺病毒载体构建方法
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
KR20210143869A (ko) * 2019-03-28 2021-11-29 더 제너럴 하스피탈 코포레이션 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터
CA3145309A1 (en) * 2019-07-10 2021-01-14 Locanabio, Inc. Rna-targeting knockdown and replacement compositions and methods for use
EP4022073A4 (en) * 2019-08-29 2023-12-20 The Board Of Trustees Of The Leland Stanford Junior University ADENO-ASSOCIATED VIRAL VECTORS TO CROSS THE HUMAN BLOOD-BRAIN BARRIER
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease
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
IL297200A (en) 2020-04-15 2022-12-01 Voyager Therapeutics Inc tau binding compounds
BR112022023106A2 (pt) 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115708866A (zh) * 2021-08-23 2023-02-24 中国科学院深圳先进技术研究院 骨源因子rankl在制备调控中枢神经系统稳态的药物或药物组合物中的应用
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023081648A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Aav衣殼變異體及其用途
WO2023235791A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024006741A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024030976A2 (en) 2022-08-03 2024-02-08 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
CN116064671A (zh) * 2022-08-10 2023-05-05 深圳先进技术研究院 一种基因递送系统将基因递送到小胶质细胞的应用
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996040954A1 (en) 1995-06-07 1996-12-19 Yale University Oral delivery of adeno-associated viral vectors
US20040076613A1 (en) 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
WO1999006562A1 (en) 1997-07-31 1999-02-11 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
JP2003523320A (ja) 1999-09-29 2003-08-05 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 ウイルスベクターの迅速なpeg改変方法、高められた遺伝子形質導入のための組成物、高められた物理的安定性を伴う組成物、およびそのための用途
WO2001036603A2 (en) 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
JP4860886B2 (ja) 2000-06-01 2012-01-25 ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二本鎖パルボウイルスベクター
AU2001268149B2 (en) 2000-06-01 2005-08-18 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
EP1402043A1 (en) 2001-07-03 2004-03-31 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
NZ600121A (en) * 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2664505T3 (es) 2001-12-17 2018-04-19 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
JP2005516949A (ja) 2001-12-19 2005-06-09 リジュン・ワン Gdnfの骨格筋へのアデノ随伴ウイルス媒介性のデリバリー
JP2005514403A (ja) * 2001-12-21 2005-05-19 ザ ソーク インスティテュート フォー バイオロジカル スタディーズ 運動ニューロンに対する標的化逆行性遺伝子送達
US6998118B2 (en) * 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
CA2520028A1 (en) * 2003-03-31 2004-10-21 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
JP2006526394A (ja) * 2003-06-03 2006-11-24 ベニテック オーストラリア リミテッド 二本鎖核酸
ES2629380T3 (es) 2003-06-19 2017-08-09 Genzyme Corporation Viriones de AAV con inmunorreactividad reducida y usos de los mismos
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
SI2292780T1 (sl) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba
WO2005037211A2 (en) * 2003-10-14 2005-04-28 Neurologix Research, Inc. Methods and compositions for the treatment of neurological disease
US8734810B2 (en) 2003-10-29 2014-05-27 Allergan, Inc. Botulinum toxin treatments of neurological and neuropsychiatric disorders
US8137960B2 (en) 2003-12-04 2012-03-20 The United States Of America As Represented By The Department Of Health And Human Services Bovine adeno-associated viral (BAAV) vector and uses thereof
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
WO2005097206A2 (en) * 2004-04-06 2005-10-20 Cedars-Sinai Medical Center Prevention and treatment of vascular disease with recombinant adeno-associated virus vectors encoding apolipoprotein a-i and apolipoprotein a-i milano
CA2568150A1 (en) * 2004-06-21 2006-01-05 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
US20060110364A1 (en) * 2004-08-20 2006-05-25 Ludwig Institute For Cancer Research Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors
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
ES2394482T3 (es) 2005-05-02 2013-02-01 Genzyme Corporation Terapia génica para trastornos de la médula espinal
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20070280906A1 (en) 2006-06-03 2007-12-06 Ognjen Petras Method to generate mirrored adenoassociated viral vectors
US20100221225A1 (en) * 2007-02-23 2010-09-02 University Of Florida Research Foundation, Inc Compositions and methods for treating glycogen storage diseases
WO2008150459A1 (en) 2007-05-30 2008-12-11 The Trustees Of The University Of Pennsylvania A method for transducing cells with primary cilia
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2192926A1 (en) 2007-10-01 2010-06-09 Alcon Research, Ltd. Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9

Also Published As

Publication number Publication date
US20180066283A1 (en) 2018-03-08
US20100240739A1 (en) 2010-09-23
LTPA2022525I1 (pt) 2022-12-12
WO2009043936A1 (en) 2009-04-09
NO2022048I1 (no) 2022-11-16
CN105087650A (zh) 2015-11-25
FR20C1057I1 (fr) 2020-12-25
CA2999607A1 (en) 2009-04-09
ES2482094T5 (es) 2024-06-03
SI2212424T1 (sl) 2014-12-31
PT3505635T (pt) 2022-08-12
CA2701561A1 (en) 2009-04-09
ES2482094T3 (es) 2014-08-01
CY1125608T1 (el) 2023-06-09
DK3505635T3 (da) 2022-08-22
JP6309373B2 (ja) 2018-04-11
EP2212424B1 (en) 2014-06-11
US11767538B2 (en) 2023-09-26
DK2212424T3 (da) 2014-07-14
SI3505635T1 (sl) 2022-10-28
EP2058401A1 (en) 2009-05-13
US9926574B2 (en) 2018-03-27
FR22C1056I1 (fr) 2023-01-06
EP2527457A1 (en) 2012-11-28
EP2212424B2 (en) 2023-11-01
PL3505635T3 (pl) 2022-09-12
EP3854878A1 (en) 2021-07-28
EP3505635A1 (en) 2019-07-03
HRP20220992T3 (hr) 2022-11-11
HUS2200047I1 (hu) 2022-12-28
PL2212424T3 (pl) 2014-11-28
ES2924892T3 (es) 2022-10-11
US20200010852A1 (en) 2020-01-09
JP2014221789A (ja) 2014-11-27
FR20C1057I2 (fr) 2024-02-16
LT3505635T (lt) 2022-09-12
HUE059864T2 (hu) 2023-01-28
EP3505635B1 (en) 2022-05-18
HRP20140643T1 (hr) 2014-11-07
PT2212424E (pt) 2014-07-25
EP2212424A1 (en) 2010-08-04
ES2727034T3 (es) 2019-10-11
FI2212424T4 (fi) 2023-12-15
JP5620819B2 (ja) 2014-11-05
JP2010540598A (ja) 2010-12-24
EP2527457B1 (en) 2019-02-20
CN101883859A (zh) 2010-11-10
CA2701561C (en) 2018-05-15

Similar Documents

Publication Publication Date Title
HUS2200047I1 (hu) Kiterjedt géntranszfer motoros neuronokhoz AAV vektorok perifériás injekciójának alkalmazásával
PT2185712T (pt) Administração de genes ao snc usando a administração periférica de vactores aav
HRP20181743T1 (hr) Poboljšani postupci za pročišćavanje rekombinantnih aav vektora
EP2342321A4 (en) CONSTRUCTION OF GENERATOR GUIDANCE BASED ON A COMPLETELY DELETED ADENOVIRUS AND USES THEREOF
EP2152316A4 (en) THERAPEUTIC DELIVERY OF INHIBITORY NUCLEIC ACID MOLECULES IN THE RESPIRATORY SYSTEM
IL199002A0 (en) Hyaluronic acid gel for intradermal injection
EP2699258A4 (en) TRANSFER OF AAV-MEDIATED CTLA-4 GENE TO TREAT THE SJÖGREN SYNDROME
EP2152883A4 (en) RECOMBINANT BACTERIA COMPRISING VECTORS FOR THE EXPRESSION OF NUCLEIC ACID SEQUENCES ENCODING ANTIGENS
EP2244824A4 (en) RECYCLING OLEFIN NAPHTHAS BY REMOVING AROMAS
EP2362909A4 (en) MAMMALIAN GENES INVOLVED IN INFECTION
EP1838856A4 (en) PROCESS FOR DELIVERING NUCLEIC ACIDS TO PERIPHERAL NEURONS
GB0818686D0 (en) Care fuel injection nozzle
IL225806A0 (en) Single exon genes encoding new bioactive peptides
AU2008905719A0 (en) Genetic vectors
GB0809627D0 (en) Targeted gene therapy
AU2009901404A0 (en) Armored repulsive personal aquatic driver
GB0814033D0 (en) Multi-cavity bioreactor chamber