Abstract: The present invention relates to conjugates formed from a cell-penetrating peptide carrier linked to a therapeutic molecule, wherein the peptide carrier is defined by specific domains and the therapeutic molecule is a nucleic acid formed of trinucleotide repeats. The present invention further relates to the use of such a conjugate in methods of treatment or as a medicament, especially in the treatment of trinucleotide repeat disorders such as myotonic dystrophy (DM1).
Type:
Application
Filed:
August 7, 2020
Publication date:
September 1, 2022
Applicants:
OXFORD UNIVERSITY INNOVATION LIMITED, UNITED KINGDOM RESEARCH AND INNOVATION, ASSOCIATION INSTITUT DE MYOLOGIE, INSERM (INSTITUT NATONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), SORBONNE UNIVERSITE
Inventors:
Matthew Wood, Miguel Varela, Ashling Holland, Richard Raz, Denis Furling, Arnaud Klein, Michael Gait