AU2019265018B2 - Methods of suppressing pathogenic mutations using programmable base editor systems - Google Patents

Methods of suppressing pathogenic mutations using programmable base editor systems

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Publication number
AU2019265018B2
AU2019265018B2 AU2019265018A AU2019265018A AU2019265018B2 AU 2019265018 B2 AU2019265018 B2 AU 2019265018B2 AU 2019265018 A AU2019265018 A AU 2019265018A AU 2019265018 A AU2019265018 A AU 2019265018A AU 2019265018 B2 AU2019265018 B2 AU 2019265018B2
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polynucleotide
base editor
domain
amino acid
protein
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AU2019265018A1 (en
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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AU2019265018A 2018-05-11 2019-05-11 Methods of suppressing pathogenic mutations using programmable base editor systems Active AU2019265018B2 (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 (2)

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AU2019265018A1 AU2019265018A1 (en) 2020-11-26
AU2019265018B2 true AU2019265018B2 (en) 2025-11-06

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US (1) US20210371858A1 (enExample)
EP (1) EP3790964A4 (enExample)
JP (2) JP7558929B2 (enExample)
KR (1) KR102959497B1 (enExample)
CN (2) CN112601816B (enExample)
AU (1) AU2019265018B2 (enExample)
CA (1) CA3100014A1 (enExample)
WO (1) WO2019217941A1 (enExample)

Families Citing this family (100)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
AU2020216484A1 (en) * 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence
CN114096666B (zh) 2019-02-13 2025-03-18 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
US20220259617A1 (en) 2019-06-13 2022-08-18 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
JP7785002B2 (ja) 2019-11-05 2025-12-12 ペアーワイズ プランツ サービシズ， インコーポレイテッド 対立遺伝子のｒｎａにコードされたｄｎａの置換のための組成物および方法
EP4093864A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
WO2021155166A1 (en) * 2020-01-31 2021-08-05 University Of Massachusetts A cas9-pdbd base editor platform with improved targeting range and specificity
US12115230B2 (en) * 2020-04-09 2024-10-15 Verve Therapeutics, Inc. Base editing of ANGPTL3 and methods of using same for treatment of disease
US20230175010A1 (en) * 2020-05-12 2023-06-08 City Of Hope Compositions and methods for base specific mitochondrial gene editing
IL298363A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
AU2021275223A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc. Coronavirus antigen compositions and their uses
EP4158032A2 (en) 2020-05-29 2023-04-05 Flagship Pioneering Innovations VI, LLC Trem compositions and methods relating thereto
US20230203510A1 (en) 2020-05-29 2023-06-29 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
IL300947A (en) 2020-09-03 2023-04-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
AU2021374974A1 (en) * 2020-11-06 2023-05-25 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
KR20230135585A (ko) 2020-12-23 2023-09-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 trem의 조성물 및 이의 용도
EP4313109A1 (en) 2021-03-31 2024-02-07 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
WO2022229903A1 (en) * 2021-04-28 2022-11-03 Genevant Sciences Gmbh Mrna delivery constructs and methods of using the same
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
KR20230016751A (ko) * 2021-07-26 2023-02-03 서울대학교산학협력단 염기 편집기 및 이의 용도
WO2023009547A1 (en) 2021-07-26 2023-02-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
AR127073A1 (es) 2021-09-17 2023-12-13 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
EP4419706A1 (en) 2021-10-18 2024-08-28 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2023096990A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovation Vi, Llc Coronavirus immunogen compositions and their uses
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
MX2024006407A (es) 2021-11-24 2024-08-19 Flagship Pioneering Innovations Vi Llc Composiciones de inmunogenos del virus varicela-zoster y sus usos.
US20250223584A2 (en) 2021-12-03 2025-07-10 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
CN118510896A (zh) 2021-12-17 2024-08-16 旗舰创业创新六公司 用于在变性条件下富集环状rna的方法
EP4453201A1 (en) 2021-12-22 2024-10-30 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
AU2022420620A1 (en) 2021-12-23 2024-07-04 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
EP4506461A4 (en) * 2022-04-06 2026-05-27 Guangzhou Reforgene Medicine Co Ltd METHOD FOR REPAIRING HBA2 GENE MUTATIONS BY SINGLE BASIC EDITING AND ITS USE
JP2025511756A (ja) 2022-04-08 2025-04-16 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー ワクチン及び関連する方法
WO2023220083A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
JP2025516638A (ja) 2022-05-13 2025-05-30 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー 二本鎖ｄｎａ組成物及び関連する方法
JP2025519070A (ja) 2022-05-17 2025-06-24 エンヴェロップ セラピューティクス， インコーポレイテッド 効率的ｉｎ ｖｉｖｏ送達のための組成物および方法
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
US20240042021A1 (en) 2022-08-01 2024-02-08 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides 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
WO2024102799A1 (en) 2022-11-08 2024-05-16 Flagship Pioneering Innovations Vi, Llc Compositions and methods for producing circular polyribonucleotides
JP2025540623A (ja) * 2022-11-11 2025-12-16 ビーム セラピューティクス インク． トランスサイレチン遺伝子の塩基編集
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
US20240238473A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. 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
EP4648794A2 (en) 2023-01-09 2025-11-19 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
TW202438515A (zh) 2023-02-06 2024-10-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 免疫調節組合物及相關方法
US20240293318A1 (en) 2023-02-13 2024-09-05 Flagship Pioneering Innovations Vii, Llc Cleavable linker-containing ionizable lipids and lipid carriers for therapeutic compositions
TW202446956A (zh) 2023-02-17 2024-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 包含經修飾的胞嘧啶的ｄｎａ組成物
KR20250151437A (ko) 2023-02-17 2025-10-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 변형된 우라실을 포함하는 dna 조성물
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
EP4680276A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
WO2024216128A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
AU2024255972A1 (en) 2023-04-12 2025-10-23 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
IL327035A (en) 2023-09-18 2026-05-01 Flagship Pioneering Innovations Vii Llc Ionizable lipidoid compositions and their medical uses
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
KR20260113064A (ko) 2023-11-14 2026-07-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 이온화 가능한 리피도이드 조성물 및 이의 치료적 용도
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
CN122349566A (zh) * 2023-12-06 2026-07-07 北京齐禾生科生物科技有限公司 优化的qbe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
US20250375499A1 (en) 2024-01-26 2025-12-11 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
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
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
TW202613158A (zh) 2024-05-16 2026-04-01 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
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
WO2026055547A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
WO2026055543A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Modified 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
WO2026102108A1 (en) 2024-11-07 2026-05-15 Flagship Pioneering Innovations Vii, Llc Dna production methods
WO2026122747A2 (en) 2024-12-05 2026-06-11 Flagship Pioneering Innovations Vii, Llc Novel immunomodulatory proteins and related methods
WO2026152023A1 (en) 2025-01-10 2026-07-16 Sail Biomedicines, Inc. Lipid compounds, lipid nanoparticle compositions and use thereof with polyribonucleotides
WO2026151879A1 (en) 2025-01-10 2026-07-16 Flagship Pioneering Innovations Vii, Llc Vaccine compositions and methods
WO2026152164A1 (en) 2025-01-13 2026-07-16 Sail Biomedicines, Inc. Cd5-targeted lipid nanoparticles encapsulating polyribonucleotides encoding chimeric antigen receptors, and methods to generate car-expressing t cells in vivo
WO2026161677A1 (en) 2025-01-24 2026-07-30 Flagship Pioneering Innovations Vii, Llc Compositions and methods
WO2026159700A1 (en) 2025-01-27 2026-07-30 Nanocell Therapeutics Holdings B.V. Methods and composition for inducing activation and dna expression in t-cells

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150166980A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Fusions of cas9 domains and nucleic acid-editing domains
WO2017093804A2 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2916499A1 (en) * 2013-07-03 2015-01-08 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
AU2015367378A1 (en) * 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2017070632A2 (en) * 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
CA3026110A1 (en) * 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US11352647B2 (en) * 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
IL264439B2 (en) * 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150166980A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Fusions of cas9 domains and nucleic acid-editing domains
WO2017093804A2 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency

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CA3100014A1 (en) 2019-11-14
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