AU2021409732A1 - Compositions and methods for reducing hla-a in a cell - Google Patents

Compositions and methods for reducing hla-a in a cell Download PDF

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Publication number
AU2021409732A1
AU2021409732A1 AU2021409732A AU2021409732A AU2021409732A1 AU 2021409732 A1 AU2021409732 A1 AU 2021409732A1 AU 2021409732 A AU2021409732 A AU 2021409732A AU 2021409732 A AU2021409732 A AU 2021409732A AU 2021409732 A1 AU2021409732 A1 AU 2021409732A1
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hla
chr6
cell
cells
engineered
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AU2021409732A9 (en
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Surbhi GOEL
Reynald Michael LESCARBEAU
Bradley Andrew MURRAY
Srijani SRIDHAR
Yong Zhang
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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    • C12N5/0636T lymphocytes
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    • A61K40/00Cellular immunotherapy
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K40/00Cellular immunotherapy
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    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
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    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/10Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the structure of the chimeric antigen receptor [CAR]
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2510/00Genetically modified cells

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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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AU2021409732A 2020-12-23 2021-12-22 Compositions and methods for reducing hla-a in a cell Pending AU2021409732A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063130095P 2020-12-23 2020-12-23
US63/130,095 2020-12-23
US202163250996P 2021-09-30 2021-09-30
US63/250,996 2021-09-30
US202163254970P 2021-10-12 2021-10-12
US63/254,970 2021-10-12
US202163288492P 2021-12-10 2021-12-10
US63/288,492 2021-12-10
PCT/US2021/064930 WO2022140586A2 (en) 2020-12-23 2021-12-22 Compositions and methods for reducing hla-a in a cell

Publications (2)

Publication Number Publication Date
AU2021409732A1 true AU2021409732A1 (en) 2023-07-20
AU2021409732A9 AU2021409732A9 (en) 2024-07-18

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AU2021409732A Pending AU2021409732A1 (en) 2020-12-23 2021-12-22 Compositions and methods for reducing hla-a in a cell

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US (1) US20240024478A1 (https=)
EP (1) EP4267724A2 (https=)
JP (1) JP2024500858A (https=)
KR (1) KR20230124664A (https=)
AU (1) AU2021409732A1 (https=)
CA (1) CA3206284A1 (https=)
CL (2) CL2023001860A1 (https=)
CO (1) CO2023009612A2 (https=)
CR (1) CR20230320A (https=)
IL (1) IL303971A (https=)
MX (1) MX2023007466A (https=)
TW (1) TW202239959A (https=)
WO (1) WO2022140586A2 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022331424A1 (en) * 2021-08-16 2024-02-22 Beam Therapeutics Inc. Persistent allogeneic modified immune cells and methods of use thereof
IL320066A (en) * 2022-10-05 2025-06-01 Garuda Therapeutics Inc Immunocompatible cells for allogeneic cell therapies to cover global, ethnic, or disease-specific populations
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
IL326191A (en) 2023-08-14 2026-03-01 Intellia Therapeutics Inc Compositions and methods for genetic modification of transforming growth factor beta receptor type 2 (tgfβr2)
WO2025049432A1 (en) * 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods for editing hla-a in cells pre-screened for the absence of one or both alleles of hla-h*01
WO2025049481A1 (en) * 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
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
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する2′修飾オリゴヌクレオチド
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのpna プローブ
EP2526199A4 (en) 2010-01-22 2013-08-07 Scripps Research Inst PROCESS FOR PRODUCING ZINC FINGERUCLEASES WITH CHANGED EFFECT
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
KR20200138445A (ko) * 2014-04-24 2020-12-09 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 입양 세포 요법 생성물을 생성하기 위한 유도 만능 줄기 세포의 응용
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
EP2990416B1 (en) 2014-08-29 2018-06-20 GEMoaB Monoclonals GmbH Universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
EP4029943A1 (en) 2017-05-08 2022-07-20 Precision Biosciences, Inc. Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
CN107723275B (zh) * 2017-10-20 2020-09-04 重庆精准生物技术有限公司 通用型car-t细胞及其制备方法和应用
WO2019147805A2 (en) 2018-01-26 2019-08-01 The Board Of Trustees Of The Leland Stanford Junior University Regulatory t cells targeted with chimeric antigen receptors
WO2019160077A1 (ja) * 2018-02-16 2019-08-22 国立大学法人京都大学 低抗原性細胞の製造方法
US11447769B2 (en) 2018-03-27 2022-09-20 The Trustees Of The University Of Pennsylvania Modified immune cells having enhanced function and methods for screening for same
AU2019282824C1 (en) 2018-06-08 2026-04-23 Intellia Therapeutics, Inc. Modified guide RNAS for gene editing
EP3581200A1 (en) 2018-06-13 2019-12-18 GEMoaB Monoclonals GmbH Reversed universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders
IL282267B2 (en) 2018-10-15 2026-03-01 Univ Massachusetts Programmed DNA editing by chimeric deaminase-nme2cas9 proteins
CN113227374A (zh) 2018-10-16 2021-08-06 因特利亚治疗公司 用于免疫疗法的组合物和方法
WO2020092057A1 (en) 2018-10-30 2020-05-07 Yale University Compositions and methods for rapid and modular generation of chimeric antigen receptor t cells
WO2020146290A1 (en) * 2019-01-07 2020-07-16 Crisp-Hr Therapeutics, Inc. A non-toxic cas9 enzyme and application thereof
CA3181340A1 (en) * 2020-04-28 2021-11-04 Intellia Therapeutics, Inc. Methods of in vitro cell delivery

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TW202239959A (zh) 2022-10-16
WO2022140586A3 (en) 2022-08-04
EP4267724A2 (en) 2023-11-01
MX2023007466A (es) 2023-08-18
KR20230124664A (ko) 2023-08-25
CA3206284A1 (en) 2022-06-30
CR20230320A (es) 2023-10-23
AU2021409732A9 (en) 2024-07-18
WO2022140586A2 (en) 2022-06-30
IL303971A (en) 2023-08-01
CL2023001860A1 (es) 2024-02-09
JP2024500858A (ja) 2024-01-10
US20240024478A1 (en) 2024-01-25
CL2023003086A1 (es) 2024-05-03
CO2023009612A2 (es) 2023-08-09

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