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1. WO2020072059 - CBLB ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE

Publication Number WO/2020/072059
Publication Date 09.04.2020
International Application No. PCT/US2018/054347
International Filing Date 04.10.2018
IPC
A61K 45/06 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
45Medicinal preparations containing active ingredients not provided for in groups A61K31/-A61K41/132
06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 31/12 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
CPC
A61K 38/465
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
465acting on ester bonds (3.1), e.g. lipases, ribonucleases
C12N 9/22
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
9Enzymes; Proenzymes; Compositions thereof
14Hydrolases (3)
16acting on ester bonds (3.1)
22Ribonucleases ; RNAses, DNAses
Applicants
  • BLUEBIRD BIO, INC. [US]/[US]
Inventors
  • JARJOUR, Jordan
  • HAVENS, Kyle
  • KROSTAG, Anne-Rachel
Agents
  • ROGEL, Mark, E.
  • CHRISTIANSEN, William T.
  • LAHERTY, Carol
  • NUDELMAN, Aaron S.
  • TEAGUE, Lawrence J.
Priority Data
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CBLB ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE
(FR) VARIANTS DE L'ENDONUCLÉASE CBLB, COMPOSITIONS ET MÉTHODES D'UTILISATION
Abstract
(EN)
The present disclosure provides improved genome editing compositions and methods for editing a CBLB gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.
(FR)
La présente invention concerne des compositions d'édition génomique améliorées et des méthodes d'édition d'un gène CBLB. L'invention concerne en outre des cellules à édition génomique pour la prévention, le traitement ou l'atténuation d'au moins un symptôme d'un cancer, d'une maladie infectieuse, d'une maladie auto-immune, d'une maladie inflammatoire ou d'une immunodéficience.
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