CA3100034A1 - Methods of editing single nucleotide polymorphism using programmable base editor systems - Google Patents

Methods of editing single nucleotide polymorphism using programmable base editor systems Download PDF

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Publication number
CA3100034A1
CA3100034A1 CA3100034A CA3100034A CA3100034A1 CA 3100034 A1 CA3100034 A1 CA 3100034A1 CA 3100034 A CA3100034 A CA 3100034A CA 3100034 A CA3100034 A CA 3100034A CA 3100034 A1 CA3100034 A1 CA 3100034A1
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Canada
Prior art keywords
cell
base editor
polynucleotide
snp
domain
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CA3100034A
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English (en)
French (fr)
Inventor
David Bryson
John Evans
Michael Packer
Jason Michael GEHRKE
Natalie PETROSSIAN
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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Publication of CA3100034A1 publication Critical patent/CA3100034A1/en
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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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
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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CA3100034A 2018-05-11 2019-05-11 Methods of editing single nucleotide polymorphism using programmable base editor systems Pending CA3100034A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862670588P 2018-05-11 2018-05-11
US62/670,588 2018-05-11
US201862780838P 2018-12-17 2018-12-17
US62/780,838 2018-12-17
US201962817986P 2019-03-13 2019-03-13
US62/817,986 2019-03-13
PCT/US2019/031898 WO2019217943A1 (en) 2018-05-11 2019-05-11 Methods of editing single nucleotide polymorphism using programmable base editor systems

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CA3100034A1 true CA3100034A1 (en) 2019-11-14

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CA3100034A Pending CA3100034A1 (en) 2018-05-11 2019-05-11 Methods of editing single nucleotide polymorphism using programmable base editor systems
CA3100037A Pending CA3100037A1 (en) 2018-05-11 2019-05-11 Methods of editing single nucleotide polymorphism using programmable base editor systems

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CA3100037A Pending CA3100037A1 (en) 2018-05-11 2019-05-11 Methods of editing single nucleotide polymorphism using programmable base editor systems

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US (2) US20210380955A1 (enExample)
EP (2) EP3790595A4 (enExample)
JP (4) JP2021523738A (enExample)
KR (4) KR102853088B1 (enExample)
CN (3) CN112469446B (enExample)
AU (3) AU2019266326B2 (enExample)
CA (2) CA3100034A1 (enExample)
WO (2) WO2019217944A1 (enExample)

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CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US11371030B2 (en) 2017-05-31 2022-06-28 The University Of Tokyo Modified Cas9 protein and use thereof
WO2019040650A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY
US20200370040A1 (en) * 2017-12-07 2020-11-26 Ramot At Tel-Aviv University Ltd. Treatment for parkinsonian patients with mutations in the lrrk2 gene
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
WO2019226953A1 (en) * 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
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
US20220127594A1 (en) * 2019-02-13 2022-04-28 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
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
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