CA3128876A1 - Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease - Google Patents

Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease Download PDF

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Publication number
CA3128876A1
CA3128876A1 CA3128876A CA3128876A CA3128876A1 CA 3128876 A1 CA3128876 A1 CA 3128876A1 CA 3128876 A CA3128876 A CA 3128876A CA 3128876 A CA3128876 A CA 3128876A CA 3128876 A1 CA3128876 A1 CA 3128876A1
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Canada
Prior art keywords
base editor
gene
adenosine
nucleic acid
snp associated
Prior art date
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Pending
Application number
CA3128876A
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English (en)
French (fr)
Inventor
Ian SLAYMAKER
Nicole GAUDELLI
Yi Yu
Bernd ZETSCHE
David A. BORN
Seung-Joo Lee
Michael Packer
Jason Michael GEHRKE
Natalie PETROSSIAN
Angelica Messana
Shaunna BERKOVITCH
David Bryson
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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Publication of CA3128876A1 publication Critical patent/CA3128876A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
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    • C12N15/113—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
    • 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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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CA3128876A 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease Pending CA3128876A1 (en)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962850919P 2019-05-21 2019-05-21
US62/850,919 2019-05-21
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 2019-05-23
US201962873138P 2019-07-11 2019-07-11
US62/873,138 2019-07-11
US201962888867P 2019-08-19 2019-08-19
US62/888,867 2019-08-19
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018073 WO2020168051A1 (en) 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

Publications (1)

Publication Number Publication Date
CA3128876A1 true CA3128876A1 (en) 2020-08-20

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Application Number Title Priority Date Filing Date
CA3128876A Pending CA3128876A1 (en) 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

Country Status (8)

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US (1) US20230140953A1 (enExample)
EP (1) EP3924484A4 (enExample)
JP (2) JP7586601B2 (enExample)
KR (1) KR20210127206A (enExample)
CN (2) CN119280261A (enExample)
AU (1) AU2020223306A1 (enExample)
CA (1) CA3128876A1 (enExample)
WO (1) WO2020168051A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023081070A1 (en) * 2021-11-02 2023-05-11 University Of Massachusetts Nme2Cas9 INLAID DOMAIN FUSION PROTEINS

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019236210B2 (en) 2018-03-14 2025-07-03 Arbor Biotechnologies, Inc. Novel CRISPR DNA targeting enzymes and systems
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12522812B2 (en) 2018-05-16 2026-01-13 Arbor Biotechnologies, Inc. CRISPR-associated systems and components
AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
CN114040977B (zh) 2019-01-31 2024-09-03 比姆医疗股份有限公司 非靶脱氨反应减低的核碱基编辑器和用于定性核碱基编辑器的测定
CA3236512C (en) 2019-02-13 2026-02-24 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
AU2020223314A1 (en) 2019-02-13 2021-08-12 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
SG11202108346WA (en) 2019-02-13 2021-08-30 Beam Therapeutics Inc Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
PH12021552613A1 (en) 2019-04-15 2022-06-27 Edigene Therapeutics Beijing Inc METHODS AND COMPOSITIONS FOR EDITING RNAs
CN114269919B (zh) 2019-07-12 2023-08-18 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑
CN115279398B (zh) 2019-09-27 2025-07-04 比姆医疗股份有限公司 治疗液态癌症的组合物和方法
PH12022551595A1 (en) * 2019-12-30 2023-11-29 Edigene Therapeutics Beijing Inc Leaper technology based method for treating mps ih and composition
BR112022015969A2 (pt) * 2020-02-13 2022-10-25 Beam Therapeutics Inc Composições e métodos para enxerto de células editadas de base
WO2022027035A1 (en) * 2020-07-27 2022-02-03 The Children's Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
US12576151B2 (en) 2020-09-25 2026-03-17 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CN116783296A (zh) * 2020-10-21 2023-09-19 小利兰·斯坦福大学托管委员会 用于募集adar的向导rna的筛选平台
WO2022221699A1 (en) * 2021-04-16 2022-10-20 Beam Therapeutics, Inc. Genetic modification of hepatocytes
CN117729931A (zh) * 2021-05-14 2024-03-19 比姆医疗股份有限公司 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法
CA3220738A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
WO2023102449A2 (en) * 2021-12-01 2023-06-08 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
CN117187220A (zh) * 2022-03-08 2023-12-08 中国科学院遗传与发育生物学研究所 腺嘌呤脱氨酶及其在碱基编辑中的用途
CN114686456B (zh) 2022-05-10 2023-02-17 中山大学 基于双分子脱氨酶互补的碱基编辑系统及其应用
JP2025530183A (ja) * 2022-09-08 2025-09-11 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ レット症候群治療法
CN121152875A (zh) * 2023-05-09 2025-12-16 北京齐禾生科生物科技有限公司 一种可作用于dna的腺苷脱氨酶及其应用
WO2024233941A1 (en) * 2023-05-11 2024-11-14 Massachusetts Eye And Ear Infirmary Base editing approaches to treat abca4-associated stargardt disease
EP4713348A1 (en) * 2023-05-15 2026-03-25 University of Georgia Research Foundation, Inc. Doubly constrained peptides as allosteric inhibitors of leucine rich repeat kinase 2
CN116836962B (zh) * 2023-06-28 2024-04-05 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器
CN120536418A (zh) * 2024-02-26 2025-08-26 尧唐(上海)生物科技有限公司 一种脱氨酶变体、包含其的碱基编辑器及其应用
WO2025184515A1 (en) * 2024-02-29 2025-09-04 Vesigen, Inc. Arrdc1-mediated microvesicle-based delivery of therapeutic agents to cells and tissues of the eye

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2591356A1 (en) * 2010-07-09 2013-05-15 Ecole Polytechnique Fédérale de Lausanne (EPFL) Method for in-vitro monitoring of neuronal disorders and use thereof
BR112017007765B1 (pt) * 2014-10-14 2023-10-03 Halozyme, Inc Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
KR20250103795A (ko) * 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
SG11201903089RA (en) * 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
WO2018213708A1 (en) * 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3064601A1 (en) * 2017-06-26 2019-01-03 President And Fellows Of Harvard College Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
CN109306361B (zh) * 2018-02-11 2022-06-28 华东师范大学 一种新的a/t到g/c碱基定点转换的基因编辑系统
CN109295186B (zh) * 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023081070A1 (en) * 2021-11-02 2023-05-11 University Of Massachusetts Nme2Cas9 INLAID DOMAIN FUSION PROTEINS

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AU2020223306A1 (en) 2021-08-05
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CN119280261A (zh) 2025-01-10
US20230140953A1 (en) 2023-05-11
CN114040970B (zh) 2024-09-27
JP7586601B2 (ja) 2024-11-19
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