KR20240025536A - X-연관 근세관성 근병증의 개선된 치료를 위한 조성물 및 방법 - Google Patents

X-연관 근세관성 근병증의 개선된 치료를 위한 조성물 및 방법 Download PDF

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KR20240025536A
KR20240025536A KR1020237044264A KR20237044264A KR20240025536A KR 20240025536 A KR20240025536 A KR 20240025536A KR 1020237044264 A KR1020237044264 A KR 1020237044264A KR 20237044264 A KR20237044264 A KR 20237044264A KR 20240025536 A KR20240025536 A KR 20240025536A
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patient
viral vector
administered
cholestasis
weeks
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Korean (ko)
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웨스톤 밀러
네이선 박텔
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아스텔라스 진 테라피스, 인크.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
    • A61K48/0033—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 non-polymeric
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K48/0083—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 administration regime
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    • 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
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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020237044264A 2021-05-24 2022-05-24 X-연관 근세관성 근병증의 개선된 치료를 위한 조성물 및 방법 Pending KR20240025536A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163192279P 2021-05-24 2021-05-24
US63/192,279 2021-05-24
US202163245611P 2021-09-17 2021-09-17
US63/245,611 2021-09-17
PCT/US2022/030716 WO2022251208A1 (en) 2021-05-24 2022-05-24 Compositions and methods for improved treatment of x-linked myotubular myopathy

Publications (1)

Publication Number Publication Date
KR20240025536A true KR20240025536A (ko) 2024-02-27

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KR1020237044264A Pending KR20240025536A (ko) 2021-05-24 2022-05-24 X-연관 근세관성 근병증의 개선된 치료를 위한 조성물 및 방법

Country Status (13)

Country Link
US (1) US20240366785A1 (https=)
EP (1) EP4351725A4 (https=)
JP (1) JP2024520411A (https=)
KR (1) KR20240025536A (https=)
AU (1) AU2022283262A1 (https=)
BR (1) BR112023024509A2 (https=)
CA (1) CA3219834A1 (https=)
CO (1) CO2023018156A2 (https=)
IL (1) IL308736A (https=)
MX (1) MX2023013926A (https=)
PH (1) PH12023553216A1 (https=)
TW (1) TW202306589A (https=)
WO (1) WO2022251208A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR125077A1 (es) * 2021-03-11 2023-06-07 Audentes Therapeutics Inc Composiciones y métodos para tratar la miopatía miotubular ligada a x

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104023727B (zh) * 2011-10-28 2017-04-05 鲁美纳医药公司 用于治疗小儿胆汁淤积性肝病的胆汁酸再循环抑制剂
US8957044B2 (en) * 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)

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IL308736A (en) 2024-01-01
CA3219834A1 (en) 2022-12-01
JP2024520411A (ja) 2024-05-24
AU2022283262A1 (en) 2023-11-23
EP4351725A1 (en) 2024-04-17
MX2023013926A (es) 2024-01-31
AU2022283262A9 (en) 2023-11-30
TW202306589A (zh) 2023-02-16
US20240366785A1 (en) 2024-11-07
WO2022251208A1 (en) 2022-12-01
EP4351725A4 (en) 2025-04-23
CO2023018156A2 (es) 2024-03-07
BR112023024509A2 (pt) 2024-02-15
PH12023553216A1 (en) 2024-04-29

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