JP2023541457A - Ｃｄ３８修飾のための化合物および方法 - Google Patents

Ｃｄ３８修飾のための化合物および方法 Download PDF

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Publication number
JP2023541457A
JP2023541457A JP2023516769A JP2023516769A JP2023541457A JP 2023541457 A JP2023541457 A JP 2023541457A JP 2023516769 A JP2023516769 A JP 2023516769A JP 2023516769 A JP2023516769 A JP 2023516769A JP 2023541457 A JP2023541457 A JP 2023541457A
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cells
grna
cell
domain
nuclease
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JPWO2022056489A5 (https=
JP2023541457A5 (https=
Inventor
ライダード，ジョン
ジョーンズ，マーク
ペティーリョ，マイケル
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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Publication of JP2023541457A5 publication Critical patent/JP2023541457A5/ja
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/24—Antigen-presenting cells [APC]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/421—Immunoglobulin superfamily
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4222—CD38 not IgG
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/99—Glycosyltransferases (2.4) transferring other glycosyl groups (2.4.99)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/02—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2) hydrolysing N-glycosyl compounds (3.2.2)
    • C12Y302/02006—NAD(P)+ nucleosidase (3.2.2.6)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/10—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the structure of the chimeric antigen receptor [CAR]
    • A61K2239/17—Hinge-spacer domain
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K2239/21—Transmembrane domain
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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
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
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JP2023516769A 2020-09-14 2021-09-14 Ｃｄ３８修飾のための化合物および方法 Abandoned JP2023541457A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063078035P 2020-09-14 2020-09-14
US63/078,035 2020-09-14
PCT/US2021/050328 WO2022056489A1 (en) 2020-09-14 2021-09-14 Compositions and methods for cd38 modification

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JP2023541457A true JP2023541457A (ja) 2023-10-02
JPWO2022056489A5 JPWO2022056489A5 (https=) 2024-09-26
JP2023541457A5 JP2023541457A5 (https=) 2024-09-26

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US (1) US20230398219A1 (https=)
EP (1) EP4211244A1 (https=)
JP (1) JP2023541457A (https=)
WO (1) WO2022056489A1 (https=)

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CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
KR20240017367A (ko) * 2021-06-02 2024-02-07 메타지노미, 인크. 클래스 ii, v형 crispr 시스템
EP4426338A2 (en) * 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Cd38 compositions and methods for immunotherapy
US20250295695A1 (en) * 2022-04-04 2025-09-25 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
CN119546757A (zh) 2022-05-25 2025-02-28 宏基因组学公司 肝酶表达的补充
KR20260036568A (ko) * 2023-07-10 2026-03-17 카스젠 라이프 사이언스 씨오., 엘티디. 세포 면역 요법용 조성물, 및 방법
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof

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EP1914242A1 (en) 2006-10-19 2008-04-23 Sanofi-Aventis Novel anti-CD38 antibodies for the treatment of cancer
DK2580243T3 (da) 2010-06-09 2020-01-13 Genmab As Antibodies against human cd38
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP3556858A3 (en) 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
AU2015358615B2 (en) 2014-12-04 2021-08-05 Janssen Biotech, Inc. Anti-CD38 antibodies for treatment of acute myeloid leukemia
EP3280803B1 (en) 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
CA2994746A1 (en) * 2015-08-11 2017-02-16 Cellectis Cells for immunotherapy engineered for targeting cd38 antigen and for cd38 gene inactivation
CN108290939B (zh) 2015-10-16 2023-01-13 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
AU2017388753A1 (en) 2016-12-30 2019-07-11 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
SG11201907870VA (en) 2017-02-28 2019-09-27 Vor Biopharma Inc Compositions and methods for inhibition of lineage specific proteins
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11718659B2 (en) 2017-08-28 2023-08-08 The Trustees Of Columbia University In The City Of New York CD33 exon 2 deficient donor stem cells for use with CD33 targeting agents
CN111918877A (zh) 2018-03-14 2020-11-10 美国卫生和人力服务部 抗cd33嵌合抗原受体及其用途
JP7575271B2 (ja) * 2018-03-29 2024-10-29 フェイト セラピューティクス，インコーポレイテッド 操作された免疫エフェクター細胞およびその使用
CA3110837A1 (en) * 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
CA3144356A1 (en) * 2019-06-21 2020-12-24 Function Oncology, Inc. A genetic pharmacopeia for comprehensive functional profiling of human cancers

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EP4211244A1 (en) 2023-07-19
US20230398219A1 (en) 2023-12-14

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