CA3106812A1 - Homology-directed repair template design and delivery to edit hemoglobin-related mutations - Google Patents

Homology-directed repair template design and delivery to edit hemoglobin-related mutations Download PDF

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Publication number
CA3106812A1
CA3106812A1 CA3106812A CA3106812A CA3106812A1 CA 3106812 A1 CA3106812 A1 CA 3106812A1 CA 3106812 A CA3106812 A CA 3106812A CA 3106812 A CA3106812 A CA 3106812A CA 3106812 A1 CA3106812 A1 CA 3106812A1
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cell
gene
nucleic acid
cells
nuclease
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CA3106812A
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English (en)
French (fr)
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David J. Rawlings
Sowmya Pattabhi
Andrew M. Scharenberg
Kyle JACOBY
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Seattle Children's Hospital
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Seattle Children's Hospital
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    • C12N15/09—Recombinant DNA-technology
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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CA3106812A 2018-04-27 2019-04-24 Homology-directed repair template design and delivery to edit hemoglobin-related mutations Pending CA3106812A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862663553P 2018-04-27 2018-04-27
US62/663,553 2018-04-27
US201962820521P 2019-03-19 2019-03-19
US62/820,521 2019-03-19
PCT/US2019/028861 WO2019209914A2 (en) 2018-04-27 2019-04-24 Homology-directed repair template design and delivery to edit hemoglobin-related mutations

Publications (1)

Publication Number Publication Date
CA3106812A1 true CA3106812A1 (en) 2019-10-31

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CA3106812A Pending CA3106812A1 (en) 2018-04-27 2019-04-24 Homology-directed repair template design and delivery to edit hemoglobin-related mutations

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US (1) US20210309995A1 (enExample)
JP (1) JP2021521855A (enExample)
KR (1) KR20210005179A (enExample)
CN (1) CN112313334A (enExample)
AU (1) AU2019261387A1 (enExample)
CA (1) CA3106812A1 (enExample)
WO (1) WO2019209914A2 (enExample)

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EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN109486814A (zh) * 2017-10-31 2019-03-19 广东赤萌医疗科技有限公司 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒
US20220251566A1 (en) * 2019-06-26 2022-08-11 The Research Foundation For The State University Of New York Cells engineered for oligonucleotide delivery, and methods for making and using thereof
CN113186203B (zh) * 2020-02-13 2022-12-30 斯微(上海)生物科技股份有限公司 治疗或者预防冠状病毒病的疫苗试剂
CN118490818A (zh) 2020-04-22 2024-08-16 生物技术欧洲股份公司 冠状病毒疫苗
EP4323513A4 (en) * 2021-04-12 2025-09-17 Kamau Therapeutics Inc METHODS AND COMPOSITIONS FOR THE PRODUCTION OF GENETICALLY MODIFIED PRIMARY CELLS
CN113174433B (zh) * 2021-04-22 2024-03-26 苏州淦江生物技术有限公司 一种基于Cas蛋白的检测方法
WO2022232839A1 (en) * 2021-04-30 2022-11-03 The Board Of Trustees Of The Leland Stanford Junior University Methods for improved production of primary cd34+ cells
CN115232811B (zh) * 2021-06-28 2025-10-28 南京启真基因工程有限公司 用于构建hbb基因突变的镰刀型细胞贫血症模型猪核移植供体细胞的方法及应用
EP4363568A4 (en) * 2021-06-29 2025-12-03 Council Scient Ind Res MODIFIED FNCAS9 AND ITS USES
CN117769650A (zh) * 2021-07-01 2024-03-26 蓝鸟生物公司 方法
AU2022352772A1 (en) * 2021-09-24 2024-04-11 Maxcyte, Inc. A method of enhanced viral transduction using electroporation
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
CN114848851A (zh) * 2022-04-29 2022-08-05 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） 治疗β-地中海贫血的药物
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
WO2025181336A1 (en) * 2024-03-01 2025-09-04 Cellectis Sa Compositions and methods for hbb-editing in hspc

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009013559A1 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
EP3981876A1 (en) * 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP4019975A1 (en) * 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes

Also Published As

Publication number Publication date
EP3784783A2 (en) 2021-03-03
WO2019209914A3 (en) 2020-01-16
AU2019261387A1 (en) 2020-11-19
WO2019209914A2 (en) 2019-10-31
KR20210005179A (ko) 2021-01-13
US20210309995A1 (en) 2021-10-07
JP2021521855A (ja) 2021-08-30
CN112313334A (zh) 2021-02-02

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