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 (enExample
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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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Publication of JP2022518931A publication Critical patent/JP2022518931A/ja
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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)
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  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
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  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Transplantation (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)

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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 (enExample) 2023-05-23

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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 (enExample)
EP (1) EP3917522A4 (enExample)
JP (2) JP2022518931A (enExample)
CN (1) CN113891712A (enExample)
AU (1) AU2020215592A1 (enExample)
BR (1) BR112021015050A2 (enExample)
CA (1) CA3127889A1 (enExample)
MX (1) MX2021009250A (enExample)
WO (1) WO2020160486A1 (enExample)

Families Citing this family (3)

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EA202192328A1 (ru) * 2019-03-22 2022-02-21 Раш Юниверсити Медикал Сентер Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления
JP2024533163A (ja) 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集
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

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Publication number Priority date Publication date Assignee Title
WO2008103993A2 (en) * 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
ES2745470T3 (es) * 2011-03-31 2020-03-02 Univ Iowa Res Found Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv
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
EP3221456B1 (en) * 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
AU2016341428B2 (en) * 2015-10-23 2021-12-02 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection

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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WO2020160486A1 (en) 2020-08-06
AU2020215592A1 (en) 2021-08-12
EP3917522A1 (en) 2021-12-08
EP3917522A4 (en) 2022-09-28
BR112021015050A2 (pt) 2021-10-05
CN113891712A (zh) 2022-01-04
MX2021009250A (es) 2021-11-04
JP2025098018A (ja) 2025-07-01
CA3127889A1 (en) 2020-08-06
US20220184188A1 (en) 2022-06-16

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