JP2024519469A5 - - Google Patents

Info

Publication number
JP2024519469A5
JP2024519469A5 JP2023566465A JP2023566465A JP2024519469A5 JP 2024519469 A5 JP2024519469 A5 JP 2024519469A5 JP 2023566465 A JP2023566465 A JP 2023566465A JP 2023566465 A JP2023566465 A JP 2023566465A JP 2024519469 A5 JP2024519469 A5 JP 2024519469A5
Authority
JP
Japan
Prior art keywords
optionally
vector
gene
aav
pcsk9
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2023566465A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024519469A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/026483 external-priority patent/WO2022232232A1/en
Publication of JP2024519469A publication Critical patent/JP2024519469A/ja
Publication of JP2024519469A5 publication Critical patent/JP2024519469A5/ja
Pending legal-status Critical Current

Links

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

Publications (2)

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

Family

ID=83847293

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023566465A Pending JP2024519469A (ja) 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

Country Status (12)

Country Link
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=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
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)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Similar Documents

Publication Publication Date Title
JP2024519469A5 (https=)
US20240360471A1 (en) Crispr/cas-related methods and compositions for treating beta hemoglobinopathies
US20240110179A1 (en) Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
US20230026726A1 (en) Crispr/cas-related methods and compositions for treating sickle cell disease
JP2022009734A (ja) タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法
EP2836226B1 (en) Compositions and methods for the treatment of hemoglobinopathies
JP2022505402A5 (https=)
JP2018516978A5 (https=)
JP7327803B2 (ja) 筋萎縮性側索硬化症（ａｌｓ）を処置する方法
JP2021511803A5 (https=)
US20240408236A1 (en) Method of modulating pcsk9 and uses thereof
JP2022505381A5 (https=)
CN114072518B (zh) 用于治疗地中海贫血或镰状细胞病的方法和组合物
CN117980482A (zh) Rbm20突变的基因组编辑
CN117561331A (zh) 用于产生经基因修饰的原代细胞的方法和组合物
US20250361505A1 (en) Compositions for and methods of gene editing
US20240335561A1 (en) A method for in vivo gene therapy to cure scd without myeloablative toxicity
KR102874560B1 (ko) 아데노연관바이러스 변이체
TW202610664A (zh) 通過表觀編輯靶向調節pcsk9的方法及其用途
CN119464292A (zh) 一种心脏特异性清除CaMKII的小型化单碱基编辑器及其应用
HK40069063B (zh) 用於治疗地中海贫血或镰状细胞病的方法和组合物
EA043766B1 (ru) Средства и способы лечения миопатий, связанных с титином, и других титинопатий