WO2024095213 - COMPOSITIONS AND METHODS FOR PREVENTING, AMELIORATING, OR TREATING SICKLE CELL DISEASE AND COMPOSITIONS AND METHODS FOR DISRUPTING GENES AND GENE SEGMENTS

National phase entry:
Publication Number WO/2024/095213
Publication Date 10.05.2024
International Application No. PCT/IB2023/061087
International Filing Date 02.11.2023
Title **
[English] COMPOSITIONS AND METHODS FOR PREVENTING, AMELIORATING, OR TREATING SICKLE CELL DISEASE AND COMPOSITIONS AND METHODS FOR DISRUPTING GENES AND GENE SEGMENTS
[French] COMPOSITIONS ET PROCÉDÉS POUR PRÉVENIR, AMÉLIORER OU TRAITER LA DRÉPANOCYTOSE ET COMPOSITION ET PROCÉDÉS POUR PERTURBER DES GÈNES ET DES SEGMENTS DE GÈNES
Applicants **
INCISIVE GENETICS, INC.
THE UNIVERSITY OF BRITISH COLUMBIA
Inventors
LEAVITT, Blair
HILL, Austin
WAGNER, Pamela
CARON, Nicholas
Priority Data
63/381,989   02.11.2022   US
63/517,518   03.08.2023   US
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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting3774
EPO Filing, Examination, Granting22154
Japan Filing, Examination, Granting2493
South Korea Filing, Examination, Granting2788
USA Filing, Examination, Granting11290
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Total: 42,499

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Abstract[English] The present disclosure provides nucleic acids, compositions and vectors containing and their use for effecting gene editing and/or gene expression alteration on sickle cell disease (SCD)-associated genes, e.g., in vivo. The present disclosure further provides compositions for and methods of effecting gene editing and/or gene expression alteration, such as on SCD-associated genes in vivo, and methods of preventing, ameliorating, or treating SCD.[French] La présente invention concerne des acides nucléiques, des compositions et des vecteurs en contenant et leur utilisation pour effectuer une édition génique et/ou une modification de l'expression génique sur des gènes associés à la drépanocytose (SCD), par exemple, in vivo. La présente invention concerne également des compositions et des procédés permettant d'effectuer une édition génique et/ou une modification de l'expression génique, afin de modifier les gènes associés à la SCD in vivo, ainsi que des procédés de prévention, d'atténuation ou de traitement de la SCD.

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