Title:
COMPOSITIONS AND METHODS FOR NON-GENOTOXIC CELL CONDITIONING
Document Type and Number:
WIPO Patent Application WO/2024/006774
Kind Code:
A3
Abstract:
Compositions and methods for non-genotoxic monoclonal antibody (mAb) conditioning, where the methods involve altering a cluster of differentiation 117 (CD117; c-KIT) polynucleotide sequence in a hematopoietic stem cell (HSC) or progenitor thereof to encode a CD117 polypeptide with reduced binding to the antibody. In various embodiments, the methods further include introducing a therapeutic alteration to a gene of the HSC or progenitor thereof for treatment of a hemoglobinopathy.
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Inventors:
GAUDELLI NICOLE (US)
BOHNUUD TANGGIS (US)
CHU SCOTT HAIHUA (US)
ZHANG KATHY YING (US)
MONDAL NANDINI (US)
EGBERT MEGAN (US)
HARTIGAN ADAM (US)
MCDONAGH CHARLOTTE (US)
BOHNUUD TANGGIS (US)
CHU SCOTT HAIHUA (US)
ZHANG KATHY YING (US)
MONDAL NANDINI (US)
EGBERT MEGAN (US)
HARTIGAN ADAM (US)
MCDONAGH CHARLOTTE (US)
Application Number:
PCT/US2023/069189
Publication Date:
March 21, 2024
Filing Date:
June 27, 2023
Export Citation:
Assignee:
BEAM THERAPEUTICS INC (US)
GAUDELLI NICOLE (US)
BOHNUUD TANGGIS (US)
GAUDELLI NICOLE (US)
BOHNUUD TANGGIS (US)
International Classes:
A61K35/28; A61K35/17; C12N15/11; C12N15/63
Domestic Patent References:
| WO2021041945A2 | 2021-03-04 | |||
| WO2020168132A1 | 2020-08-20 |
Foreign References:
| US20210261955A1 | 2021-08-26 |
Other References:
YAN RENHONG; WANG RUOKE; JU BIN; YU JINFANG; ZHANG YUANYUAN; LIU NAN; WANG JIA; ZHANG QI; CHEN PENG; ZHOU BING; LI YANING; SHEN YA: "Structural basis for bivalent binding and inhibition of SARS-CoV-2 infection by human potent neutralizing antibodies", CELL RESEARCH, SPRINGER SINGAPORE, SINGAPORE, vol. 31, no. 5, 17 March 2021 (2021-03-17), Singapore , pages 517 - 525, XP037441887, ISSN: 1001-0602, DOI: 10.1038/s41422-021-00487-9
DATABASE PROTEIN 10 February 2021 (2021-02-10), ANONYMOUS: "immunoglobulin variable region, partial [Homo sapiens]", XP093152620, Database accession no. QRG33535.1
DATABASE PROTEIN 23 July 2016 (2016-07-23), ANONYMOUS: "immunoglobulin light chain, partial [Homo sapiens]", XP093152621, Database accession no. BAF64543.1
DATABASE PROTEIN 20 May 2022 (2022-05-20), ANONYMOUS: "KIT proto-oncogene receptor tyrosine kinase transcript variant 1 [Homo sapiens]", XP093152622, Database accession no. UQV80935.1
DATABASE PROTEIN 21 July 2005 (2005-07-21), ANONYMOUS: "KIT protein [Homo sapiens]", XP093152625, Database accession no. AAH71593.1
DATABASE PROTEIN 7 December 2019 (2019-12-07), ANONYMOUS: "TadA*-T7RNAP fusion protein (plasmid) [Expression vector pSEVA221TadA*-T7RNAP]", XP093152630, Database accession no. QGQ59661.1
DATABASE PROTEIN 10 February 2021 (2021-02-10), ANONYMOUS: "immunoglobulin variable region, partial [Homo sapiens]", XP093152620, Database accession no. QRG33535.1
DATABASE PROTEIN 23 July 2016 (2016-07-23), ANONYMOUS: "immunoglobulin light chain, partial [Homo sapiens]", XP093152621, Database accession no. BAF64543.1
DATABASE PROTEIN 20 May 2022 (2022-05-20), ANONYMOUS: "KIT proto-oncogene receptor tyrosine kinase transcript variant 1 [Homo sapiens]", XP093152622, Database accession no. UQV80935.1
DATABASE PROTEIN 21 July 2005 (2005-07-21), ANONYMOUS: "KIT protein [Homo sapiens]", XP093152625, Database accession no. AAH71593.1
DATABASE PROTEIN 7 December 2019 (2019-12-07), ANONYMOUS: "TadA*-T7RNAP fusion protein (plasmid) [Expression vector pSEVA221TadA*-T7RNAP]", XP093152630, Database accession no. QGQ59661.1
Attorney, Agent or Firm:
HUNTER-ENSOR, Melissa (US)
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