JP2024534387A - 造血細胞におけるマルチプレックス塩基編集のための組成物及び方法 - Google Patents

造血細胞におけるマルチプレックス塩基編集のための組成物及び方法 Download PDF

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JP2024534387A
JP2024534387A JP2024516579A JP2024516579A JP2024534387A JP 2024534387 A JP2024534387 A JP 2024534387A JP 2024516579 A JP2024516579 A JP 2024516579A JP 2024516579 A JP2024516579 A JP 2024516579A JP 2024534387 A JP2024534387 A JP 2024534387A
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grna
cell
siglec
grnas
gene
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JP2024534387A5 (enExample
Inventor
ライダード，ジョン
ファリャ，アレハンドラ
パイク，エリザベス
ヘイゼルベイカー，デーン
チャクラボーティ，ティルタ
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Vor Biopharma Inc
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Vor Biopharma Inc
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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
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
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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
    • C12N9/14—Hydrolases (3)
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12Y—ENZYMES
    • 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)
    • C12Y305/04002—Adenine deaminase (3.5.4.2)
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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)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)

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  • Animal Behavior & Ethology (AREA)
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  • Plant Pathology (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2024516579A 2021-09-14 2022-09-14 造血細胞におけるマルチプレックス塩基編集のための組成物及び方法 Pending JP2024534387A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163244219P 2021-09-14 2021-09-14
US63/244,219 2021-09-14
US202163278375P 2021-11-11 2021-11-11
US63/278,375 2021-11-11
US202263343408P 2022-05-18 2022-05-18
US63/343,408 2022-05-18
PCT/US2022/043557 WO2023043858A1 (en) 2021-09-14 2022-09-14 Compositions and methods for multiplex base editing in hematopoietic cells

Publications (2)

Publication Number Publication Date
JP2024534387A true JP2024534387A (ja) 2024-09-20
JP2024534387A5 JP2024534387A5 (enExample) 2025-09-25

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JP2024516579A Pending JP2024534387A (ja) 2021-09-14 2022-09-14 造血細胞におけるマルチプレックス塩基編集のための組成物及び方法

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US (1) US20240408234A1 (enExample)
EP (1) EP4401744A4 (enExample)
JP (1) JP2024534387A (enExample)
AU (1) AU2022345079A1 (enExample)
CA (1) CA3232593A1 (enExample)
WO (1) WO2023043858A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3110837A1 (en) * 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
WO2024227082A1 (en) * 2023-04-26 2024-10-31 Vijay Sankaran Base-editing perturbation screens and uses thereof
WO2024238565A1 (en) 2023-05-15 2024-11-21 Vor Biopharma Inc. Egf-like module containing mucin-like hormone-like 2 (erm2) binding agents and methods of use thereof
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025126049A2 (en) * 2023-12-11 2025-06-19 Crispr Therapeutics Ag Multiplex gene editing
WO2026077659A1 (en) * 2024-09-17 2026-04-16 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through epigenome editing

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016337525B2 (en) * 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
CA3110837A1 (en) * 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
AU2020338011A1 (en) * 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
WO2021081244A1 (en) * 2019-10-22 2021-04-29 Fred Hutchinson Cancer Research Center Base editor-mediated cd33 reduction to selectively protect therapeutic cells
CN116157135A (zh) * 2020-06-03 2023-05-23 纽约市哥伦比亚大学理事会 使用基于crispr的碱基编辑器系统抑制谱系特异性抗原的组合物和方法

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CA3232593A1 (en) 2023-03-23
US20240408234A1 (en) 2024-12-12
EP4401744A4 (en) 2026-03-18
WO2023043858A1 (en) 2023-03-23
AU2022345079A1 (en) 2024-04-04
EP4401744A1 (en) 2024-07-24

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