WO2025238578 - GUIDE RNAS FOR ALLELE-SELECTIVE GENE EDITING, AND COMPOSITIONS AND METHODS RELATING THERETO

National phase entry is expected:
Publication Number WO/2025/238578
Publication Date 20.11.2025
International Application No. PCT/IB2025/055061
International Filing Date 14.05.2025
Title **
[English] GUIDE RNAS FOR ALLELE-SELECTIVE GENE EDITING, AND COMPOSITIONS AND METHODS RELATING THERETO
[French] ARN GUIDE POUR L'ÉDITION GÉNÉTIQUE SÉLECTIVE D'ALLÈLES, COMPOSITIONS ET PROCÉDÉS ASSOCIÉS
Applicants **
INCISIVE GENETICS, INC.
THE UNIVERSITY OF BRITISH COLUMBIA
Inventors
CARON, Nicholas
CAPRON, Arnaud
HILL, Austin
WAGNER, Pamela
LEAVITT, Blair
CHENG, Jung-Chien
LIU, Yaxin
YI, Yuyin
Priority Data
63/647,377   14.05.2024   US
63/680,656   08.08.2024   US
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译文

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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting3884
EPO Filing, Examination, Granting22042
Japan Filing, Examination, Granting2431
South Korea Filing, Examination, Granting2768
USA Filing, Examination, Granting10490
MasterCard Visa
Total: 41,615
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Abstract[English] The present disclosure provides gRNAs for allele-selective gene editing, nucleic acids encoding such a gRNA, vectors comprising such a nucleic acid, RNPs comprising such a gRNA and a Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition using such gRNAs. The present disclosure further provides lipid-based TCVs.[French] La présente invention concerne des ARNg pour l'édition génétique sélective d'allèles, des acides nucléiques codant pour un tel ARNg, des vecteurs comportant un tel acide nucléique, des RNP comportant un tel ARNg et une protéine Cas, des compositions pharmaceutiques comportant l'un quelconque des éléments précités, et des kits comportant l'un quelconque des éléments précités. La présente invention concerne également des procédés liés à ces ARNg, notamment des procédés de fabrication, des procédés permettant d'effectuer une édition génétique sélective des allèles, et des procédés permettant de prévenir, traiter ou atténuer un symptôme d'une maladie, d'un trouble ou d'une pathologie cible à l'aide de ces ARNg. La présente invention concerne également des TCV à base de lipides.