JP2024519469A - 遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法 - Google Patents

遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法 Download PDF

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JP2024519469A
JP2024519469A JP2023566465A JP2023566465A JP2024519469A JP 2024519469 A JP2024519469 A JP 2024519469A JP 2023566465 A JP2023566465 A JP 2023566465A JP 2023566465 A JP2023566465 A JP 2023566465A JP 2024519469 A JP2024519469 A JP 2024519469A
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vector
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pcsk9
aav
sequence
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ワン，リリー
ウイルソン，ジェームス・エム
トレティアコバ，アンナ
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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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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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JP2023566465A 2021-04-27 2022-04-27 遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法 Pending JP2024519469A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202163180603P 2021-04-27 2021-04-27
US63/180,603 2021-04-27
US202163242474P 2021-09-09 2021-09-09
US63/242,474 2021-09-09
US202163244205P 2021-09-14 2021-09-14
US63/244,205 2021-09-14
US202263301933P 2022-01-21 2022-01-21
US63/301,933 2022-01-21
US202263331385P 2022-04-15 2022-04-15
US63/331,385 2022-04-15
PCT/US2022/026483 WO2022232232A1 (en) 2021-04-27 2022-04-27 Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders

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JP2024519469A true JP2024519469A (ja) 2024-05-14
JP2024519469A5 JP2024519469A5 (https=) 2025-05-07

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US (1) US20240197916A1 (https=)
EP (1) EP4330412A4 (https=)
JP (1) JP2024519469A (https=)
KR (1) KR20240001708A (https=)
AU (1) AU2022266662A1 (https=)
BR (1) BR112023021129A2 (https=)
CA (1) CA3216285A1 (https=)
CO (1) CO2023016031A2 (https=)
IL (1) IL307958A (https=)
MX (1) MX2023012747A (https=)
TW (1) TW202304528A (https=)
WO (1) WO2022232232A1 (https=)

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Publication number Priority date Publication date Assignee Title
WO2023140971A1 (en) * 2022-01-21 2023-07-27 The Trustees Of The University Ofpennsylvania Methods for treatment of ornithine transcarbamylase (otc) deficiency
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
KR20260027890A (ko) * 2023-04-28 2026-03-03 오스페달레 산 라파엘 에스.알.엘. 벡터 생산
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
CN116790604B (zh) * 2023-08-18 2023-10-27 成都中科奥格生物科技有限公司 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途
WO2025217184A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of phenylketonuria (pku)
WO2025217186A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of citrullinemia type 1 (ctln1)

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EP3288594B1 (en) * 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EP3416689B1 (en) * 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
EP3429632B1 (en) * 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
US20180127786A1 (en) * 2016-09-23 2018-05-10 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
IL301059B2 (en) * 2017-04-21 2026-04-01 Prec Biosciences Inc Engineered nucleosides specific for recognition sequences in the PCSK9 gene
CA3079428A1 (en) * 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

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MX2023012747A (es) 2024-01-05
US20240197916A1 (en) 2024-06-20
CO2023016031A2 (es) 2023-12-11
WO2022232232A1 (en) 2022-11-03
EP4330412A4 (en) 2026-01-28
CA3216285A1 (en) 2022-11-03
AU2022266662A1 (en) 2023-11-02
AU2022266662A9 (en) 2023-11-16
IL307958A (en) 2023-12-01
EP4330412A1 (en) 2024-03-06
TW202304528A (zh) 2023-02-01
KR20240001708A (ko) 2024-01-03
BR112023021129A2 (pt) 2023-12-12

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