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1. WO2021247856 - COMPOSITIONS AND METHODS FOR INHIBITION OF LINEAGE SPECIFIC ANTIGENS USING CRISPR-BASED BASE EDITOR SYSTEMS

Publication Number WO/2021/247856
Publication Date 09.12.2021
International Application No. PCT/US2021/035691
International Filing Date 03.06.2021
IPC
A61K 35/12 2015.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
A61K 35/28 2015.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
C12N 5/0789 2010.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
078Cells from blood or from the immune system
0789Stem cells; Multipotent progenitor cells
Applicants
  • THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK [US]/[US]
Inventors
  • MUKHERJEE, Siddhartha
  • BOROT, Florence
  • ALI, Abdullah Mahmood
Agents
  • KRAMER CARNEY, Bonnie
Priority Data
63/033,96603.06.2020US
63/033,97003.06.2020US
63/183,79104.05.2021US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR INHIBITION OF LINEAGE SPECIFIC ANTIGENS USING CRISPR-BASED BASE EDITOR SYSTEMS
(FR) COMPOSITIONS ET PROCÉDÉS D'INHIBITION D'ANTIGÈNES SPÉCIFIQUES DE LA LIGNÉE À L'AIDE DE SYSTÈMES D'ÉDITEUR DE BASE À BASE DE CRISPR
Abstract
(EN) Disclosed herein are methods of administering an agent targeting a lineage- specific cell-surface antigen, e.g., CD33 or EMR2, and a population of hematopoietic cells that are altered in the expression of the lineage- specific cell- surface antigen, e.g., CD33 or EMR2, for immunotherapy of hematological malignancies. Also disclosed herein are methods of administering an agent targeting more than one lineage-specific cell- surface antigen, and a population of hematopoietic cells that are altered in the expression of more than one lineage- specific cell-surface antigen, for immunotherapy of hematological malignancies. Cells comprising mutations in CD33, or EMR2, or more than one lineage- specific cell-surface antigen are also provided, as are methods of producing such cells using CRISPR-based base editor systems.
(FR) L'invention concerne des procédés d'administration d'un agent ciblant un antigène de surface cellulaire spécifique à une lignée, par exemple CD33 ou EMR2, et une population de cellules hématopoïétiques qui sont modifiées en ce qui concerne l'expression de l'antigène de surface cellulaire spécifique de la lignée, par exemple CD33 ou EMR2, pour l'immunothérapie de malignités hématologiques. L'invention concerne également des procédés d'administration d'un agent ciblant un ou plusieurs antigènes de surface cellulaire spécifiques à une lignée et une population de cellules hématopoïétiques qui sont modifiées en ce qui concerne l'expression d'antigènes de surface cellulaire spécifiques de la lignée, pour l'immunothérapie de malignités hématologiques. L'invention concerne également des cellules comprenant des mutations dans CD33, ou EMR2, ou plus d'un antigène de surface cellulaire spécifique d'une lignée, ainsi que des procédés de production de telles cellules à l'aide de systèmes d'éditeur de base à base de CRISPR.
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