JP2024546578A - ムコ多糖症ｉｉｉａ型の治療のための遺伝子療法 - Google Patents

ムコ多糖症ｉｉｉａ型の治療のための遺伝子療法 Download PDF

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JP2024546578A
JP2024546578A JP2024528581A JP2024528581A JP2024546578A JP 2024546578 A JP2024546578 A JP 2024546578A JP 2024528581 A JP2024528581 A JP 2024528581A JP 2024528581 A JP2024528581 A JP 2024528581A JP 2024546578 A JP2024546578 A JP 2024546578A
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hsgsh
raav
nucleic acid
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JPWO2023086928A5 (https=
JP2024546578A5 (https=
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オルドー，ジュリエット
ウイルソン，ジェームス・エム
ラッスーリ－テイラー，レイダ
ドー，フン
タスケ，スティーブン
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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
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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
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    • 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)
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • 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
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    • C12Y—ENZYMES
    • C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/102—Vectors comprising a special translation-regulating system regulates levels of translation inhibiting translation

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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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JP2024528581A 2021-11-12 2022-11-11 ムコ多糖症ｉｉｉａ型の治療のための遺伝子療法 Pending JP2024546578A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163278775P 2021-11-12 2021-11-12
US63/278,775 2021-11-12
US202163288293P 2021-12-10 2021-12-10
US63/288,293 2021-12-10
PCT/US2022/079701 WO2023086928A2 (en) 2021-11-12 2022-11-11 Gene therapy for treatment of mucopolysaccharidosis iiia

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JP2024546578A true JP2024546578A (ja) 2024-12-26
JPWO2023086928A5 JPWO2023086928A5 (https=) 2025-11-18
JP2024546578A5 JP2024546578A5 (https=) 2025-11-18

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JP2024528581A Pending JP2024546578A (ja) 2021-11-12 2022-11-11 ムコ多糖症ｉｉｉａ型の治療のための遺伝子療法

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US (1) US20250041445A1 (https=)
EP (1) EP4430201A4 (https=)
JP (1) JP2024546578A (https=)
KR (1) KR20240133693A (https=)
AU (1) AU2022388800A1 (https=)
CA (1) CA3237987A1 (https=)
CO (1) CO2024007283A2 (https=)
IL (1) IL312676A (https=)
MX (1) MX2024005735A (https=)
WO (1) WO2023086928A2 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019013528A (es) * 2017-05-12 2020-07-14 The Children´S Hospital Of Philadelphia Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia).
KR102823833B1 (ko) * 2017-11-30 2025-06-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 뮤코다당류증 iiia형에 대한 유전자 요법
AR116624A1 (es) * 2018-10-10 2021-05-26 Amicus Therapeutics Inc Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso
US12454701B2 (en) * 2018-12-05 2025-10-28 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN114127275A (zh) * 2019-04-30 2022-03-01 宾夕法尼亚州大学信托人 可用于治疗庞贝病的组合物

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US20250041445A1 (en) 2025-02-06
CO2024007283A2 (es) 2024-07-18
EP4430201A2 (en) 2024-09-18
CA3237987A1 (en) 2023-05-19
WO2023086928A2 (en) 2023-05-19
AU2022388800A1 (en) 2024-06-27
WO2023086928A3 (en) 2023-06-22
KR20240133693A (ko) 2024-09-04
MX2024005735A (es) 2024-12-06
EP4430201A4 (en) 2026-04-08
IL312676A (en) 2024-07-01

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