CN113891712A - 用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法 - Google Patents

用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法 Download PDF

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Publication number
CN113891712A
CN113891712A CN202080024872.5A CN202080024872A CN113891712A CN 113891712 A CN113891712 A CN 113891712A CN 202080024872 A CN202080024872 A CN 202080024872A CN 113891712 A CN113891712 A CN 113891712A
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aav
dose
tpp1
vector genome
vector
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Pending
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CN202080024872.5A
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English (en)
Chinese (zh)
Inventor
大卫·威廉·安德森
玛丽安·托托
苏·E·I·达森
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Spark Therapeutics Inc
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Spark Therapeutics Inc
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    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • 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
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Transplantation (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
CN202080024872.5A 2019-02-01 2020-01-31 用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法 Pending CN113891712A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962800131P 2019-02-01 2019-02-01
US62/800,131 2019-02-01
PCT/US2020/016235 WO2020160486A1 (en) 2019-02-01 2020-01-31 Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2

Publications (1)

Publication Number Publication Date
CN113891712A true CN113891712A (zh) 2022-01-04

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ID=71841678

Family Applications (1)

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CN202080024872.5A Pending CN113891712A (zh) 2019-02-01 2020-01-31 用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法

Country Status (9)

Country Link
US (1) US20220184188A1 (https=)
EP (1) EP3917522A4 (https=)
JP (2) JP2022518931A (https=)
CN (1) CN113891712A (https=)
AU (1) AU2020215592A1 (https=)
BR (1) BR112021015050A2 (https=)
CA (1) CA3127889A1 (https=)
MX (1) MX2021009250A (https=)
WO (1) WO2020160486A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3132379A1 (en) * 2019-03-22 2020-10-01 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal storage disorders
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100221225A1 (en) * 2007-02-23 2010-09-02 University Of Florida Research Foundation, Inc Compositions and methods for treating glycogen storage diseases
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
CN105764532A (zh) * 2013-07-26 2016-07-13 衣阿华大学研究基金会 治疗脑疾病的方法和组合物
CN108601771A (zh) * 2015-10-23 2018-09-28 衣阿华大学研究基金会 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US10900053B2 (en) * 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
CA3041548A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100221225A1 (en) * 2007-02-23 2010-09-02 University Of Florida Research Foundation, Inc Compositions and methods for treating glycogen storage diseases
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
CN105764532A (zh) * 2013-07-26 2016-07-13 衣阿华大学研究基金会 治疗脑疾病的方法和组合物
CN108601771A (zh) * 2015-10-23 2018-09-28 衣阿华大学研究基金会 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MARCO A PASSINI等: "Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis", J NEUROSCI, 1 February 2006 (2006-02-01) *

Also Published As

Publication number Publication date
JP2025098018A (ja) 2025-07-01
CA3127889A1 (en) 2020-08-06
JP2022518931A (ja) 2022-03-17
MX2021009250A (es) 2021-11-04
WO2020160486A1 (en) 2020-08-06
US20220184188A1 (en) 2022-06-16
AU2020215592A1 (en) 2021-08-12
EP3917522A4 (en) 2022-09-28
BR112021015050A2 (pt) 2021-10-05
EP3917522A1 (en) 2021-12-08

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Application publication date: 20220104