KR20230003012A - 유전자 요법을 위한 아스파르토아실라아제(aspa)를 인코딩하는 변형된 핵산 및 벡터 - Google Patents

유전자 요법을 위한 아스파르토아실라아제(aspa)를 인코딩하는 변형된 핵산 및 벡터 Download PDF

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KR20230003012A
KR20230003012A KR1020227041079A KR20227041079A KR20230003012A KR 20230003012 A KR20230003012 A KR 20230003012A KR 1020227041079 A KR1020227041079 A KR 1020227041079A KR 20227041079 A KR20227041079 A KR 20227041079A KR 20230003012 A KR20230003012 A KR 20230003012A
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South Korea
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nucleic acid
seq
acid sequence
vector
aspa
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Korean (ko)
Inventor
파올라 레오네
제레미 프란시스
바젤 타리크 아사프
쇼 아사노
캐서린 헤일즈
앨리슨 피. 버그
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화이자 인코포레이티드
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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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    • 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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    • C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
    • C12Y305/01015—Aspartoacylase (3.5.1.15)
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
KR1020227041079A 2020-04-28 2021-04-22 유전자 요법을 위한 아스파르토아실라아제(aspa)를 인코딩하는 변형된 핵산 및 벡터 Pending KR20230003012A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063016507P 2020-04-28 2020-04-28
US63/016,507 2020-04-28
US202063077144P 2020-09-11 2020-09-11
US63/077,144 2020-09-11
PCT/US2021/028658 WO2021221995A1 (en) 2020-04-28 2021-04-22 Modified nucleic acids encoding aspartoacylase (aspa) and vector for gene therapy

Publications (1)

Publication Number Publication Date
KR20230003012A true KR20230003012A (ko) 2023-01-05

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227041079A Pending KR20230003012A (ko) 2020-04-28 2021-04-22 유전자 요법을 위한 아스파르토아실라아제(aspa)를 인코딩하는 변형된 핵산 및 벡터

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Country Link
US (1) US20230165977A1 (https=)
EP (1) EP4142759A4 (https=)
JP (1) JP7821742B2 (https=)
KR (1) KR20230003012A (https=)
CN (1) CN115461066B (https=)
AU (1) AU2021263534A1 (https=)
BR (1) BR112022021964A2 (https=)
CA (1) CA3174070A1 (https=)
IL (1) IL297605A (https=)
TW (1) TW202206599A (https=)
WO (1) WO2021221995A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3234809A1 (en) 2021-10-20 2023-04-27 Steven Goldman Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss
EP4426331A1 (en) 2021-11-02 2024-09-11 University of Rochester Tcf7l2 mediated remyelination in the brain
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025129157A1 (en) * 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL156752A0 (en) 2001-01-06 2004-02-08 Gen Hospital Corp Gene for identifying individuals with familial dysautonomia
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
EP3364997B1 (en) * 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US10610606B2 (en) * 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof

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Publication number Publication date
US20230165977A1 (en) 2023-06-01
JP7821742B2 (ja) 2026-02-27
CN115461066B (zh) 2026-01-06
BR112022021964A2 (pt) 2023-03-14
EP4142759A4 (en) 2024-05-29
WO2021221995A1 (en) 2021-11-04
AU2021263534A1 (en) 2023-01-19
CA3174070A1 (en) 2021-11-04
JP2023524024A (ja) 2023-06-08
EP4142759A1 (en) 2023-03-08
CN115461066A (zh) 2022-12-09
IL297605A (en) 2022-12-01
TW202206599A (zh) 2022-02-16

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