KR20230029922A - 피루베이트 키나아제 결핍증(pkd) 유전자 편집 치료 방법 - Google Patents

피루베이트 키나아제 결핍증(pkd) 유전자 편집 치료 방법 Download PDF

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KR20230029922A
KR20230029922A KR1020237003103A KR20237003103A KR20230029922A KR 20230029922 A KR20230029922 A KR 20230029922A KR 1020237003103 A KR1020237003103 A KR 1020237003103A KR 20237003103 A KR20237003103 A KR 20237003103A KR 20230029922 A KR20230029922 A KR 20230029922A
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South Korea
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seq
sequence
cells
corpk
aav
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KR1020237003103A
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Korean (ko)
Inventor
산츠 호세 카를로스 세고비아
버스타만테 오스카 친타나
바케로 사라 파나나스
매튜 포르테우스
Original Assignee
센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피.
콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드
푼다시안 인스티튜토 데 인베스티카시온 사니타리아 푼다시안 시메네스 디아즈
더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Application filed by 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피., 콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드, 푼다시안 인스티튜토 데 인베스티카시온 사니타리아 푼다시안 시메네스 디아즈, 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티, 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 filed Critical 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피.
Publication of KR20230029922A publication Critical patent/KR20230029922A/ko
Pending legal-status Critical Current

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    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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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
    • A61K48/005—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
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12Y207/0104—Pyruvate kinase (2.7.1.40)

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020237003103A 2020-06-26 2021-06-28 피루베이트 키나아제 결핍증(pkd) 유전자 편집 치료 방법 Pending KR20230029922A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20382568.2 2020-06-26
EP20382568.2A EP3929294A1 (en) 2020-06-26 2020-06-26 Pyruvate kinase deficiency (pkd) gene editing treatment method
PCT/EP2021/067719 WO2021260227A1 (en) 2020-06-26 2021-06-28 Pyruvate kinase deficiency (pkd) gene editing treatment method

Publications (1)

Publication Number Publication Date
KR20230029922A true KR20230029922A (ko) 2023-03-03

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KR1020237003103A Pending KR20230029922A (ko) 2020-06-26 2021-06-28 피루베이트 키나아제 결핍증(pkd) 유전자 편집 치료 방법

Country Status (9)

Country Link
US (1) US20230303990A1 (https=)
EP (2) EP3929294A1 (https=)
JP (1) JP7847778B2 (https=)
KR (1) KR20230029922A (https=)
CN (1) CN116648511A (https=)
AU (1) AU2021298186A1 (https=)
CA (1) CA3188164A1 (https=)
MX (1) MX2023000035A (https=)
WO (1) WO2021260227A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4534654A1 (en) * 2023-10-02 2025-04-09 Consorcio Centro de Investigación Biomédica en Red Pre-stimulation method of cd34+ cells to achieve clinically relevant gene editing frequencies

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MX2023000035A (es) 2023-04-10
WO2021260227A1 (en) 2021-12-30
EP4172332A1 (en) 2023-05-03
JP7847778B2 (ja) 2026-04-20
JP2023531718A (ja) 2023-07-25
CA3188164A1 (en) 2021-12-30
US20230303990A1 (en) 2023-09-28
AU2021298186A1 (en) 2023-02-09
CN116648511A (zh) 2023-08-25
EP3929294A1 (en) 2021-12-29

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Patent event date: 20230126

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Patent event code: PA02012R01D

Patent event date: 20240621

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