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1. WO2021044379 - UNIVERSAL DONOR CELLS

Publication Number WO/2021/044379
Publication Date 11.03.2021
International Application No. PCT/IB2020/058281
International Filing Date 04.09.2020
IPC
C12N 5/10 2006.01
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
10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C12N 15/11 2006.01
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
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
A61K 35/12 2015.01
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
CPC
A61K 35/39
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
37Digestive system
39Pancreas; Islets of Langerhans
C07K 14/70532
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
70503Immunoglobulin superfamily
70532B7 molecules, e.g. CD80, CD86
C07K 14/70539
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
70503Immunoglobulin superfamily
70539MHC-molecules, e.g. HLA-molecules
C07K 2319/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
02containing a signal sequence
C07K 2319/03
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
03containing a transmembrane segment
C07K 2319/09
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
09containing a nuclear localisation signal
Applicants
  • CRISPR THERAPEUTICS AG [CH]/[CH]
Inventors
  • REZANIA, Alireza
  • RAMOS-ZAYAS, Rebeca
Agents
  • VOSSIUS & PARTNER
Priority Data
62/896,47305.09.2019US
62/979,77121.02.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) UNIVERSAL DONOR CELLS
(FR) CELLULES DONNEUSES UNIVERSELLES
Abstract
(EN)
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
(FR)
L'invention concerne des cellules génétiquement modifiées compatibles avec de multiples sujets, par exemple des cellules donneuses universelles, et des procédés de génération desdites cellules génétiquement modifiées. Les cellules donneuses universelles comprennent au moins une modification génétique à l'intérieur ou à proximité d'au moins un gène codant un facteur de survie, la modification génétique comprenant une insertion d'un polynucléotide codant pour un facteur tolérogène. Les cellules donneuses universelles peuvent en outre comprendre au moins une modification génétique à l'intérieur ou à proximité d'un gène codant un ou plusieurs antigènes leucocytaires humains MHC-I ou CMH-II ou un composant ou un régulateur transcriptionnel d'un complexe MHC-I ou CMH-II, ladite modification génétique comprenant une insertion d'un polynucléotide codant pour un second facteur tolérogène.
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