AU2018383712B2 - Cpf1-related methods and compositions for gene editing - Google Patents

Cpf1-related methods and compositions for gene editing

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Publication number
AU2018383712B2
AU2018383712B2 AU2018383712A AU2018383712A AU2018383712B2 AU 2018383712 B2 AU2018383712 B2 AU 2018383712B2 AU 2018383712 A AU2018383712 A AU 2018383712A AU 2018383712 A AU2018383712 A AU 2018383712A AU 2018383712 B2 AU2018383712 B2 AU 2018383712B2
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Australia
Prior art keywords
ascpf1
bcl11a
trac
ciita
sequence
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AU2018383712A
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AU2018383712A1 (en
Inventor
Jennifer GORI
Hariharan JAYARAM
John Zuris
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Editas Medicine Inc
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Editas Medicine Inc
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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AU2018383712A 2017-12-11 2018-12-11 Cpf1-related methods and compositions for gene editing Active AU2018383712B2 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201762597118P 2017-12-11 2017-12-11
US62/597,118 2017-12-11
US201862623501P 2018-01-29 2018-01-29
US62/623,501 2018-01-29
US201862664905P 2018-04-30 2018-04-30
US62/664,905 2018-04-30
US201862746494P 2018-10-16 2018-10-16
US62/746,494 2018-10-16
PCT/US2018/065032 WO2019118516A1 (en) 2017-12-11 2018-12-11 Cpf1-related methods and compositions for gene editing

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AU2018383712B2 true AU2018383712B2 (en) 2025-09-04

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EP (1) EP3724326A1 (enExample)
JP (2) JP2021505187A (enExample)
KR (1) KR20200097760A (enExample)
CN (1) CN111712569A (enExample)
AU (1) AU2018383712B2 (enExample)
CA (1) CA3085338A1 (enExample)
MX (1) MX2020006072A (enExample)
WO (1) WO2019118516A1 (enExample)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
SG11202008956XA (en) * 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US20200216825A1 (en) * 2019-01-08 2020-07-09 Integrated Dna Technologies, Inc. CAS12a MUTANT GENES AND POLYPEPTIDES ENCODED BY SAME
AU2020223060B2 (en) * 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
EP4047087A4 (en) * 2019-08-19 2023-08-23 Southern Medical University Construction of high-fidelity crispr/ascpf1 mutant and application thereof
JP7696335B2 (ja) * 2019-09-09 2025-06-20 スクライブ・セラピューティクス・インコーポレイテッド 免疫療法に使用するための組成物および方法
US20230009232A1 (en) * 2019-11-20 2023-01-12 Cartherics Pty. Ltd. Method for providing immune cells with enhanced function
CN111647618A (zh) * 2020-01-15 2020-09-11 温州医科大学 一种新型基因组编辑工具(Lb2Cas12a-RVR)及其构建方法和应用方法
CN113355389B (zh) * 2020-03-05 2022-11-15 广西扬翔股份有限公司 利用CRISPR/Cas12a系统靶向富集核酸目标区域的方法及其应用
EP4146800A1 (en) * 2020-05-08 2023-03-15 Metagenomi, Inc. Enzymes with ruvc domains
KR20220005208A (ko) * 2020-07-06 2022-01-13 주식회사 지씨셀 면역원성이 감소된 신규한 이식용 세포
KR102497690B1 (ko) * 2020-09-22 2023-02-10 (주)지플러스생명과학 신규한 crispr 연관 단백질 및 이의 용도
US20230374478A1 (en) * 2020-09-22 2023-11-23 G Flas Life Sciences Modified cas12a protein and use thereof
US20230416732A1 (en) * 2020-10-30 2023-12-28 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting bcl11a and uses thereof
AU2021369840A1 (en) * 2020-10-30 2023-06-01 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting b2m and uses thereof
US20240026351A1 (en) * 2020-10-30 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting trac and uses thereof
WO2022103935A1 (en) * 2020-11-11 2022-05-19 The Trustees Of Columbia University In The City Of New York Multiplex epigenome editing
US11591381B2 (en) 2020-11-30 2023-02-28 Crispr Therapeutics Ag Gene-edited natural killer cells
AU2021392032A1 (en) 2020-12-03 2023-06-22 Century Therapeutics, Inc. Genetically engineered cells and uses thereof
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
AU2021396403A1 (en) * 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
EP4259160A4 (en) * 2020-12-14 2025-05-14 Emendobio Inc. BIALLELIC INACTIVATION OF B2M
KR20230135068A (ko) * 2020-12-23 2023-09-22 인텔리아 테라퓨틱스, 인크. 세포에서 ciita를 유전적으로 변형시키기 위한 조성물및 방법
EP4271798A1 (en) 2020-12-30 2023-11-08 CRISPR Therapeutics AG Compositions and methods for differentiating stem cells into nk cells
CN112725487A (zh) * 2021-02-03 2021-04-30 张国良 用于链霉素耐药结核分枝杆菌的核酸快速检测试剂盒及其检测方法
US20240200059A1 (en) 2021-04-09 2024-06-20 Vor Biopharma Inc. Photocleavable guide rnas and methods of use thereof
WO2022236147A1 (en) * 2021-05-06 2022-11-10 Artisan Development Labs, Inc. Modified nucleases
KR102787489B1 (ko) * 2021-06-16 2025-03-31 한국생명공학연구원 enCpf1 및 키메릭 DNA-RNA 가이드를 포함하는 유전체 교정 또는 발현 억제용 조성물
US20230016422A1 (en) * 2021-06-23 2023-01-19 Crispr Therapeutics Ag Engineered cells with improved protection from natural killer cell killing
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
WO2023004411A1 (en) * 2021-07-23 2023-01-26 Icahn School Of Medicine At Mount Sinai A method for in vivo gene therapy to cure scd without myeloablative toxicity
US20250122534A1 (en) 2021-08-02 2025-04-17 Vor Biopharma Inc. Compositions and methods for gene modification
WO2023028348A1 (en) 2021-08-27 2023-03-02 Metagenomi, Inc. Enzymes with ruvc domains
US20240417755A1 (en) 2021-09-27 2024-12-19 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
CN116144631B (zh) * 2023-01-17 2023-09-15 华中农业大学 耐热型核酸内切酶及其介导的基因编辑系统
US20240254463A1 (en) * 2023-02-01 2024-08-01 Vedabio, Inc. Engineered stable nucleic acid-guided nucleases
CN116179513B (zh) * 2023-03-10 2023-12-22 之江实验室 一种Cpf1蛋白及其在基因编辑中的应用
CN116179511B (zh) * 2023-03-10 2023-12-22 之江实验室 Cpf1蛋白在制备用于核酸检测的试剂盒中的应用
WO2024249346A1 (en) 2023-05-26 2024-12-05 Editas Medicine, Inc. Crispr-related methods and compositions targeting cd70 expression
WO2024249342A1 (en) 2023-05-26 2024-12-05 Editas Medicine, Inc. Crispr-related methods and compositions targeting ptpn2 expression
WO2024249348A2 (en) 2023-05-26 2024-12-05 Editas Medicine, Inc. Crispr-related methods and compositions targeting fl1-1 expression

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017106657A1 (en) * 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2017189308A1 (en) * 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6080569A (en) * 1993-06-24 2000-06-27 Merck & Co., Inc. Adenovirus vectors generated from helper viruses and helper-dependent vectors
US5994627A (en) 1995-03-31 1999-11-30 Common Wealth Scientific And Industrial Research Organisation Genetic sequences conferring nematode resistance in plants and uses therefor
CA2329947A1 (en) * 1998-06-09 1999-12-16 Merck & Co., Inc. Novel adenoviral vectors for gene therapy
WO2009131632A1 (en) * 2008-04-14 2009-10-29 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
GB201013153D0 (en) 2010-08-04 2010-09-22 Touchlight Genetics Ltd Primer for production of closed linear DNA
BR112014027813A2 (pt) 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
US9540659B2 (en) * 2012-07-06 2017-01-10 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
EP2970943A4 (en) * 2013-03-15 2017-04-19 The Children's Hospital of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
US10494645B2 (en) * 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP4427809A3 (en) * 2014-10-31 2024-12-04 The Trustees of The University of Pennsylvania Altering gene expression in car-t cells and uses thereof
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
US20160362667A1 (en) * 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
WO2017011519A1 (en) * 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
GB2557123B (en) * 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
SG11201807538PA (en) * 2016-03-04 2018-09-27 Editas Medicine Inc Crispr-cpf1-related methods, compositions and components for cancer immunotherapy
IL313038A (en) * 2016-03-14 2024-07-01 Editas Medicine Inc Methods and preparations related to CRISPR/CAS - for the treatment of diseases in the hemoglobin cell
SG11201809144XA (en) * 2016-04-18 2018-11-29 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
KR102424476B1 (ko) * 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
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
WO2019084168A1 (en) * 2017-10-24 2019-05-02 Editas Medicine, Inc. SYSTEMS AND METHODS FOR TREATING HYPER-IGM SYNDROME
EP3707266A1 (en) * 2017-11-07 2020-09-16 Editas Medicine, Inc. Targeted integration systems and methods for the treatment of hemoglobinopathies

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017106657A1 (en) * 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2017189308A1 (en) * 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems

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