CA3100014A1 - Methods of suppressing pathogenic mutations using programmable base editor systems - Google Patents

Methods of suppressing pathogenic mutations using programmable base editor systems Download PDF

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Publication number
CA3100014A1
CA3100014A1 CA3100014A CA3100014A CA3100014A1 CA 3100014 A1 CA3100014 A1 CA 3100014A1 CA 3100014 A CA3100014 A CA 3100014A CA 3100014 A CA3100014 A CA 3100014A CA 3100014 A1 CA3100014 A1 CA 3100014A1
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Prior art keywords
polynucleotide
cell
domain
base editor
protein
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CA3100014A
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English (en)
French (fr)
Inventor
John Evans
Yanfang FU
Michael Packer
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
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    • C12N2310/00—Structure or type of the nucleic acid
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CA3100014A 2018-05-11 2019-05-11 Methods of suppressing pathogenic mutations using programmable base editor systems Pending CA3100014A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862670498P 2018-05-11 2018-05-11
US62/670,498 2018-05-11
US201862780864P 2018-12-17 2018-12-17
US62/780,864 2018-12-17
PCT/US2019/031896 WO2019217941A1 (en) 2018-05-11 2019-05-11 Methods of suppressing pathogenic mutations using programmable base editor systems

Publications (1)

Publication Number Publication Date
CA3100014A1 true CA3100014A1 (en) 2019-11-14

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ID=68468436

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CA3100014A Pending CA3100014A1 (en) 2018-05-11 2019-05-11 Methods of suppressing pathogenic mutations using programmable base editor systems

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US (1) US20210371858A1 (https=)
EP (1) EP3790964A4 (https=)
JP (2) JP7558929B2 (https=)
CN (2) CN119570787A (https=)
AU (1) AU2019265018B2 (https=)
CA (1) CA3100014A1 (https=)
WO (1) WO2019217941A1 (https=)

Families Citing this family (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
WO2020160517A1 (en) * 2019-01-31 2020-08-06 Beam Therapeutics Inc. Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence
CA3128755C (en) 2019-02-13 2024-06-04 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
JP7759311B2 (ja) 2019-07-19 2025-10-23 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー リコンビナーゼ組成物及び使用方法
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
WO2021151073A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
WO2021155166A1 (en) * 2020-01-31 2021-08-05 University Of Massachusetts A cas9-pdbd base editor platform with improved targeting range and specificity
MX2022012683A (es) * 2020-04-09 2023-01-11 Verve Therapeutics Inc Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades.
US20230175010A1 (en) * 2020-05-12 2023-06-08 City Of Hope Compositions and methods for base specific mitochondrial gene editing
EP4153223A1 (en) 2020-05-20 2023-03-29 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
IL298362A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Coronavirus antigen compositions and their uses
WO2021243301A2 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
KR20230029685A (ko) 2020-05-29 2023-03-03 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Trem 조성물 및 이에 관련된 방법
CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
AU2021336976A1 (en) 2020-09-03 2023-03-23 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20220145334A1 (en) * 2020-11-06 2022-05-12 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
IL303886A (en) 2020-12-23 2023-08-01 Flagship Pioneering Inc Compositions of modified trems and uses thereof
EP4313109A1 (en) 2021-03-31 2024-02-07 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
US20240216545A1 (en) * 2021-04-28 2024-07-04 Genevant Sciences Gmbh Mrna delivery constructs and methods of using the same
AU2022272250A1 (en) * 2021-05-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating transthyretin amyloidosis
WO2022246266A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
EP4377457A1 (en) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
MX2024003262A (es) 2021-09-17 2024-06-19 Flagship Pioneering Innovations Vi Llc Composiciones y metodos para producir poliribonucleotidos circulares.
CA3235625A1 (en) 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
EP4436984A1 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Coronavirus immunogen compositions and their uses
CN118401544A (zh) 2021-11-24 2024-07-26 旗舰创业创新六公司 水痘-带状疱疹病毒免疫原组合物及其用途
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
EP4441074A2 (en) 2021-12-03 2024-10-09 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
KR20240118174A (ko) 2021-12-17 2024-08-02 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변성 조건 하에서의 원형 rna의 풍부화를 위한 방법
EP4453201A1 (en) 2021-12-22 2024-10-30 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
KR20240118881A (ko) 2021-12-23 2024-08-05 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 항푸소제닉 폴리펩티드를 인코딩하는 원형 폴리리보뉴클레오티드
WO2023196634A2 (en) 2022-04-08 2023-10-12 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
CA3257006A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc TREM COMPOSITIONS AND METHODS OF USE FOR TREATING PROLIFERATIVE DISORDERS
TW202409283A (zh) 2022-05-13 2024-03-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 雙股ｄｎａ組合物及相關方法
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
US20250179492A1 (en) 2022-06-22 2025-06-05 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
EP4565604A2 (en) 2022-08-01 2025-06-11 Flagship Pioneering Innovations VII, LLC Immunomodulatory proteins and related methods
WO2024035952A1 (en) 2022-08-12 2024-02-15 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
US20240174732A1 (en) 2022-10-05 2024-05-30 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additional polypeptides and their use in treating cancer
EP4612296A1 (en) 2022-10-31 2025-09-10 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
TW202434728A (zh) 2022-11-08 2024-09-01 美商旗艦先鋒創新有限責任公司 用於產生環狀多核糖核苷酸的組成物和方法
EP4615425A1 (en) * 2022-11-11 2025-09-17 Beam Therapeutics Inc. Base editing of transthyretin gene
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於將治療劑遞送至骨之組成物和方法
US20240252520A1 (en) 2023-01-09 2024-08-01 Beth Israel Deaconess Medical Center, Inc. Therapeutic agents and their use for treating chronic wounds
WO2024151673A2 (en) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Recombinant nucleic acid molecules and their use in wound healing
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
WO2024151583A2 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
WO2024167885A1 (en) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Immunomodulatory compositions and related methods
CN121263425A (zh) 2023-02-13 2026-01-02 旗舰创业创新第七有限责任公司 含可裂解接头的可电离脂质和用于治疗性组合物的脂质载体
IL322378A (en) 2023-02-17 2025-09-01 Flagship Pioneering Innovations Vii Llc DNA compositions containing modified cytosine
EP4665405A1 (en) 2023-02-17 2025-12-24 Flagship Pioneering Innovations VII, LLC Dna compositions comprising modified uracil
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
CN121358494A (zh) 2023-03-15 2026-01-16 旗舰创业创新第六有限责任公司 免疫原性组合物及其用途
EP4695391A1 (en) 2023-04-12 2026-02-18 Flagship Pioneering Innovations VI, LLC Trems for use in correction of missense mutations
EP4695393A1 (en) 2023-04-12 2026-02-18 Flagship Pioneering Innovations VI, LLC Modified trems, compositions, and related methods thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
KR20260044217A (ko) 2023-07-25 2026-04-01 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 Cas 엔도뉴클레아제 및 관련 방법
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025054236A2 (en) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025064475A2 (en) 2023-09-18 2025-03-27 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025106670A1 (en) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025119306A1 (zh) * 2023-12-06 2025-06-12 北京齐禾生科生物科技有限公司 优化工程化t细胞的碱基编辑系统及其应用
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025158420A1 (en) 2024-01-25 2025-07-31 Nanocell Therapeutics Holdings B.V. Methods and composition for inducing activation and dna expression in t-cells
TW202545974A (zh) 2024-01-26 2025-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 免疫受體抑制蛋白及相關方法
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026055543A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Modified dna compositions and related methods
WO2026055547A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
WO2026064313A1 (en) 2024-09-17 2026-03-26 Flagship Pioneering Innovations Vii, Llc Rna compositions and related methods
WO2026085300A1 (en) 2024-10-16 2026-04-23 Flagship Pioneering Innovations Vii, Llc Compositions and methods

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9458457B2 (en) * 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3234134B1 (en) * 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
AU2017257274B2 (en) * 2016-04-19 2023-07-13 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
IL308824A (en) * 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
EP4485466A3 (en) * 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
CN110352244B (zh) * 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
CN106916852B (zh) * 2017-04-13 2020-12-04 上海科技大学 一种碱基编辑系统及其构建和应用方法

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