WO2025226841 - GENE THERAPY APPROACH FOR TREATING DISORDERS ASSOCIATED WITH TNNT2

National phase entry is expected:
Publication Number WO/2025/226841
Publication Date 30.10.2025
International Application No. PCT/US2025/026015
International Filing Date 23.04.2025
Title **
[English] GENE THERAPY APPROACH FOR TREATING DISORDERS ASSOCIATED WITH TNNT2
[French] APPROCHE DE THÉRAPIE GÉNIQUE POUR LE TRAITEMENT DE TROUBLES ASSOCIÉS À TNNT2
Applicants **
KATE THERAPEUTICS, INC.
Inventors
FIELDEN, Mark
TABEBORDBAR, Mohammadsharif
Priority Data
63/638,086   24.04.2024   US
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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting2489
EPO Filing, Examination, Granting15851
Japan Filing, Examination, Granting2618
South Korea Filing, Examination, Granting3015
USA Filing, Examination, Granting7340
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Total: 31,313
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Abstract[English] The present invention provides expression cassettes and methods that reduce expression of an endogenous TNNT2 gene while providing a non-mutant TNNT2 transgene (tTNNT2), resulting in greatly increased TNNT2 transgene incorporation in the sarcomere than from delivery of the TNNT2 transgene alone.[French] La présente invention concerne des cassettes d'expression et des procédés qui réduisent l'expression d'un gène TNNT2 endogène tout en fournissant un transgène TNNT2 non mutant (tTNNT2), ce qui entraîne une incorporation nettement plus importante du transgène TNNT2 dans le sarcomère que lors de l'administration du transgène TNNT2 seul.