CN115715185A - 用于治疗神经系统疾病的组合物和方法 - Google Patents

用于治疗神经系统疾病的组合物和方法 Download PDF

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Publication number
CN115715185A
CN115715185A CN202180042428.0A CN202180042428A CN115715185A CN 115715185 A CN115715185 A CN 115715185A CN 202180042428 A CN202180042428 A CN 202180042428A CN 115715185 A CN115715185 A CN 115715185A
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raav
subject
nucleic acid
viral vector
sequence
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Pending
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CN202180042428.0A
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English (en)
Chinese (zh)
Inventor
R·M·贝利
S·J·格雷
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University of Texas System
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University of Texas System
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—DNA 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202180042428.0A 2020-04-15 2021-04-14 用于治疗神经系统疾病的组合物和方法 Pending CN115715185A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063010179P 2020-04-15 2020-04-15
US63/010,179 2020-04-15
PCT/US2021/027266 WO2021211700A1 (en) 2020-04-15 2021-04-14 Compositions and methods for treatment of neurological disorders

Publications (1)

Publication Number Publication Date
CN115715185A true CN115715185A (zh) 2023-02-24

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CN202180042428.0A Pending CN115715185A (zh) 2020-04-15 2021-04-14 用于治疗神经系统疾病的组合物和方法

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US (2) US11753655B2 (https=)
EP (1) EP4135661A4 (https=)
JP (1) JP2023522883A (https=)
KR (1) KR20230004617A (https=)
CN (1) CN115715185A (https=)
AU (1) AU2021255934A1 (https=)
CA (1) CA3175406A1 (https=)
IL (1) IL296857A (https=)
WO (1) WO2021211700A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11753655B2 (en) * 2020-04-15 2023-09-12 The Board Of Regents Of The University Of Texas System Compositions and methods for treatment of neurological disorders
WO2023102518A1 (en) * 2021-12-03 2023-06-08 The Board Of Regents Of The University Of Texas System Gnao1 gene therapy vectors and uses thereof
EP4713462A1 (en) * 2023-05-18 2026-03-25 Gencorrex Therapies Inc. Solute carrier family 6 member 8 vectors and methods for use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
CN108348528A (zh) * 2015-09-17 2018-07-31 科达生物治疗公司 治疗神经障碍的组合物和方法

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Publication number Priority date Publication date Assignee Title
US8676330B2 (en) * 2009-03-20 2014-03-18 ElectroCore, LLC Electrical and magnetic stimulators used to treat migraine/sinus headache and comorbid disorders
US20070292922A1 (en) * 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
EP2771367B1 (en) * 2011-10-25 2017-12-06 Indiana University Research and Technology Corporation Gigaxonin fusion protein and methods for treating giant axonal neuropathy
US10016514B2 (en) * 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
WO2018109207A1 (en) * 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
US11753655B2 (en) * 2020-04-15 2023-09-12 The Board Of Regents Of The University Of Texas System Compositions and methods for treatment of neurological disorders

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
CN108348528A (zh) * 2015-09-17 2018-07-31 科达生物治疗公司 治疗神经障碍的组合物和方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RACHEL M. BAILEY ET AL.: "Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy", METHODS ＆ CLINICAL DEVELOPMENT, vol. 9, 7 March 2018 (2018-03-07), pages 161 *

Also Published As

Publication number Publication date
WO2021211700A1 (en) 2021-10-21
US12534741B2 (en) 2026-01-27
JP2023522883A (ja) 2023-06-01
CA3175406A1 (en) 2021-10-21
AU2021255934A1 (en) 2022-10-20
US20240102050A1 (en) 2024-03-28
KR20230004617A (ko) 2023-01-06
EP4135661A4 (en) 2024-07-10
IL296857A (en) 2022-11-01
EP4135661A1 (en) 2023-02-22
US11753655B2 (en) 2023-09-12
US20210324411A1 (en) 2021-10-21

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