JP2022518931A - 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 - Google Patents

遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 Download PDF

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JP2022518931A
JP2022518931A JP2021544194A JP2021544194A JP2022518931A JP 2022518931 A JP2022518931 A JP 2022518931A JP 2021544194 A JP2021544194 A JP 2021544194A JP 2021544194 A JP2021544194 A JP 2021544194A JP 2022518931 A JP2022518931 A JP 2022518931A
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aav
dose
tpp1
vector
vector genome
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JP2022518931A5 (https=
Inventor
デイビッド ウィリアム アンダーソン，
メアリーアン トト，
スー イー．アイ． ダーセン，
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Spark Therapeutics Inc
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Spark Therapeutics Inc
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Priority to JP2025030051A priority Critical patent/JP2025098018A/ja
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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)
JP2021544194A 2019-02-01 2020-01-31 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 Pending JP2022518931A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025030051A JP2025098018A (ja) 2019-02-01 2025-02-27 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法

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

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JP2025030051A Division JP2025098018A (ja) 2019-02-01 2025-02-27 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法

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JP2022518931A true JP2022518931A (ja) 2022-03-17
JP2022518931A5 JP2022518931A5 (https=) 2023-05-23

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JP2021544194A Pending JP2022518931A (ja) 2019-02-01 2020-01-31 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法
JP2025030051A Withdrawn JP2025098018A (ja) 2019-02-01 2025-02-27 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法

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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)

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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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016530246A (ja) * 2013-07-26 2016-09-29 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 脳疾患を処置するための方法および組成物
WO2018085688A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2132309A4 (en) * 2007-02-23 2011-01-05 Univ Florida COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
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
KR102738860B1 (ko) * 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016530246A (ja) * 2013-07-26 2016-09-29 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 脳疾患を処置するための方法および組成物
WO2018085688A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BIODRUGS, vol. 31, JPN6024043745, 2017, pages 317 - 334, ISSN: 0005447271 *
DISCOVERY MEDICINE, vol. 19, no. 102, JPN6024000994, 2015, pages 49 - 57, ISSN: 0005447268 *
GENE THERAPY, vol. 14, JPN6024043746, 2007, pages 989 - 997, ISSN: 0005447272 *
MINGOZZI F. ET AL.: "Overcoming Pre-Existing Humoral Immunity to AAV Using Capsid Decoys", MOLECULAR THERAPY, vol. Vol.21, Suppl.1, JPN6024000995, 2013, pages 45 - 110, ISSN: 0005447270 *
SCI TRANSL MED (AUTHOR MANUSCRIPT), vol. Vol.5, No.194 194ra92, JPN6024000996, 2013, pages 1 - 20, ISSN: 0005447269 *

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Publication number Publication date
JP2025098018A (ja) 2025-07-01
CN113891712A (zh) 2022-01-04
CA3127889A1 (en) 2020-08-06
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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