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1. WO2021207710 - BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE

Publication Number WO/2021/207710
Publication Date 14.10.2021
International Application No. PCT/US2021/026729
International Filing Date 09.04.2021
IPC
C12N 15/11 2006.1
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
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
Applicants
  • VERVE THERAPEUTICS, INC. [US]/[US]
Inventors
  • CHADWICK, Alexandra
  • RAJEEV, Kallanthottathil G.
  • ROHDE, Ellen
  • CHENG, Christopher
  • REISS, Caroline
Agents
  • WANG, Yingjiao
Priority Data
63/007,79709.04.2020US
63/007,80309.04.2020US
63/045,03226.06.2020US
63/045,03326.06.2020US
63/136,08711.01.2021US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
(FR) ÉDITION DE BASE DE ANGPTL3 ET PROCÉDÉS D'UTILISATION DE CELLE-CI POUR LE TRAITEMENT D'UNE MALADIE
Abstract
(EN)
Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.
(FR)
L'invention concerne des compositions pour la modification ou l'édition de gènes et des procédés d'utilisation de celles-ci pour traiter ou prévenir certaines conditions. L'invention concerne des compositions et des procédés spécifiques capables d'éditer de manière sûre et efficace des cibles géniques exprimées dans le foie pour réduire durablement le cholestérol LDL, ce qui permet de traiter une cause principale de maladie cardiovasculaire.
Latest bibliographic data on file with the International Bureau