RU2751953C2 - Модифицированные капсидные белки для улучшения доставки парвовирусных векторов - Google Patents

Модифицированные капсидные белки для улучшения доставки парвовирусных векторов Download PDF

Info

Publication number
RU2751953C2
RU2751953C2 RU2018125937A RU2018125937A RU2751953C2 RU 2751953 C2 RU2751953 C2 RU 2751953C2 RU 2018125937 A RU2018125937 A RU 2018125937A RU 2018125937 A RU2018125937 A RU 2018125937A RU 2751953 C2 RU2751953 C2 RU 2751953C2
Authority
RU
Russia
Prior art keywords
capsid protein
capsid
amino acid
parvovirus
viral vector
Prior art date
Application number
RU2018125937A
Other languages
English (en)
Russian (ru)
Other versions
RU2018125937A3 (enExample
RU2018125937A (ru
Inventor
Ричард Джуд САМУЛЬСКИ
Джейми ВАРИСХАЛЬК
Original Assignee
Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл
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 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл filed Critical Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл
Publication of RU2018125937A publication Critical patent/RU2018125937A/ru
Publication of RU2018125937A3 publication Critical patent/RU2018125937A3/ru
Application granted granted Critical
Publication of RU2751953C2 publication Critical patent/RU2751953C2/ru

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14021—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
RU2018125937A 2015-12-14 2016-12-14 Модифицированные капсидные белки для улучшения доставки парвовирусных векторов RU2751953C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562266941P 2015-12-14 2015-12-14
US62/266,941 2015-12-14
PCT/US2016/066466 WO2017106236A1 (en) 2015-12-14 2016-12-14 Modified capsid proteins for enhanced delivery of parvovirus vectors

Publications (3)

Publication Number Publication Date
RU2018125937A RU2018125937A (ru) 2020-01-20
RU2018125937A3 RU2018125937A3 (enExample) 2020-05-14
RU2751953C2 true RU2751953C2 (ru) 2021-07-21

Family

ID=59057532

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2018125937A RU2751953C2 (ru) 2015-12-14 2016-12-14 Модифицированные капсидные белки для улучшения доставки парвовирусных векторов

Country Status (12)

Country Link
US (3) US11124546B2 (enExample)
EP (2) EP4659816A3 (enExample)
JP (3) JP7406783B2 (enExample)
CN (2) CN116478254A (enExample)
AU (2) AU2016371779C1 (enExample)
CA (1) CA3008013A1 (enExample)
DK (1) DK3390429T3 (enExample)
ES (1) ES3040934T3 (enExample)
MX (2) MX395524B (enExample)
PL (1) PL3390429T3 (enExample)
RU (1) RU2751953C2 (enExample)
WO (1) WO2017106236A1 (enExample)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
US11124546B2 (en) * 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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.
WO2019191716A1 (en) 2018-03-29 2019-10-03 University Of Florida Research Foundation, Incorporated Aav6 variants
EP3781187A4 (en) * 2018-03-29 2021-12-22 Asklepios Biopharmaceutical, Inc. TROPICAL RECOMBINANT AAV6 LIVER VECTORS TO Bypass NEUTRALIZATION
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019221992A1 (en) 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
AU2020263392A1 (en) * 2019-04-26 2021-10-28 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
AU2020299026A1 (en) * 2019-07-04 2022-01-20 Children's Medical Research Institute Methods and AAV vectors for in vivo transduction
EP4010357A2 (en) 2019-08-09 2022-06-15 University of Florida Research Foundation, Incorporated Aav capsid variants for targeting human glioblastoma cells
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
EP4055030A4 (en) 2019-11-08 2024-05-22 President And Fellows Of Harvard College VIRAL CAPSID POLYPEPTIDES
EP4055034A4 (en) 2019-11-08 2024-05-29 President And Fellows Of Harvard College VIRAL CAPSID POLYPEPTIDES
CN110950934B (zh) * 2019-12-31 2022-11-04 复旦大学 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
US20230374542A1 (en) 2020-10-07 2023-11-23 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21)
AU2021410043A1 (en) * 2020-12-23 2023-06-22 Synteny Therapeutics, Inc. Protoparvovirus and tetraparvovirus compositions and methods for gene therapy
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
EP4623087A2 (en) * 2022-11-21 2025-10-01 Shape Therapeutics Inc. Systems for amplification of aav cap protein
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
EP4695396A1 (en) 2023-04-10 2026-02-18 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
US20260234665A1 (en) * 2023-04-17 2026-08-13 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced intra-articular delivery of adenovirus-associated virus (aav) vectors
CN121311595A (zh) 2023-04-17 2026-01-09 阿斯克生物公司 转基因在灵长类动物中可调节的受控表达
EP4786601A1 (en) 2025-01-31 2026-08-05 AskBio Inc. Helper plasmids and uses thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
IL122206A0 (en) 1995-06-07 1998-04-05 Univ North Carolina Adeno-associated virus vectors for gene expression
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
US7071172B2 (en) 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
JP2009535339A (ja) * 2006-04-28 2009-10-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
EP2396343B1 (en) * 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
KR102298579B1 (ko) * 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
EP3049527A4 (en) * 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
IL314171A (en) * 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
US11124546B2 (en) * 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
WO2007089632A3 (en) * 2006-01-27 2008-12-11 Univ North Carolina Heparin and heparan sulfate binding chimeric vectors

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ФИЛЛИПОВИЧ Ю.Б., Биохимические основы жизнедеятельности человека, ВЛАДОС, 2005, стр. 49,50. PAKULA A.A., SAUER R.T., Genetic analysis of protein stability and function, Annual review of genetics, 1989, vol.23, pp.289-310; стр. 305-306. *

Also Published As

Publication number Publication date
US20190382452A1 (en) 2019-12-19
JP7406783B2 (ja) 2023-12-28
JP2022065003A (ja) 2022-04-26
CA3008013A1 (en) 2017-06-22
AU2021203779A1 (en) 2021-07-08
EP4659816A3 (en) 2025-12-31
AU2016371779A1 (en) 2018-07-19
CN108602859B (zh) 2023-05-26
EP3390429A1 (en) 2018-10-24
CN116478254A (zh) 2023-07-25
WO2017106236A1 (en) 2017-06-22
NZ783341A (en) 2025-05-02
PL3390429T3 (pl) 2025-10-20
MX395524B (es) 2025-03-25
JP2024045227A (ja) 2024-04-02
AU2016371779B2 (en) 2021-04-01
US11124546B2 (en) 2021-09-21
AU2021203779B2 (en) 2022-08-18
NZ743916A (en) 2025-03-28
EP4659816A2 (en) 2025-12-10
MX2018007237A (es) 2018-12-11
US12319715B2 (en) 2025-06-03
US20210403510A1 (en) 2021-12-30
MX2022006652A (es) 2022-07-05
EP3390429B1 (en) 2025-06-18
RU2018125937A3 (enExample) 2020-05-14
CN108602859A (zh) 2018-09-28
AU2016371779C1 (en) 2022-10-13
JP2019506445A (ja) 2019-03-07
RU2018125937A (ru) 2020-01-20
US20250333453A1 (en) 2025-10-30
DK3390429T3 (da) 2025-08-25
ES3040934T3 (en) 2025-11-05
BR112018011881A2 (pt) 2018-12-04
EP3390429A4 (en) 2019-07-17

Similar Documents

Publication Publication Date Title
US12319715B2 (en) Modified capsid proteins for enhanced delivery of parvovirus vectors
US11939597B2 (en) Restrictive inverted terminal repeats for viral vectors
EP2673289B1 (en) Viral vectors with modified transduction profiles and methods of making and using the same
US20230013145A1 (en) Aav capsid-promoter interactions and cell selective gene expression
US20240271115A1 (en) Methods and compositions for depleting antibodies
US20220347315A1 (en) Methods and compositions for increasing transduction efficiency with cell membrane fusion proteins
US20240238448A1 (en) Use of chemical epigenetic modifiers to modulate gene expression from vectors
HK40097682A (en) Modified capsid proteins for enhanced delivery of parvovirus vectors
HK1260937B (en) Modified capsid proteins for enhanced delivery of parvovirus vectors
HK1260937A1 (en) Modified capsid proteins for enhanced delivery of parvovirus vectors
HK1192565B (en) Viral vectors with modified transduction profiles and methods of making and using the same
HK1192565A (en) Viral vectors with modified transduction profiles and methods of making and using the same