Genethon’s Optidys project is rewarded in response to the call for proposals “Innovation in Biotherapies and Bioproduction” led by BPIfrance as part of France 2030.
Driven by Isabelle Richard, Head of Progressive Muscular Dystrophies team, and Sonia Albini, researcher in her team, Optidys aims to develop a gene therapy product for Duchenne muscular dystrophy characterized by better efficacy, safety and persistence over time and aimed at all patients regardless of their genetic mutation.
“This funding gives us the opportunity to confirm and optimize the efficacy of the gene therapy product we have developed, based on the generation of a therapeutic gene that is more complete and more efficient than the one currently used in clinical trials, combined with a new vector developed to more specifically target skeletal and cardiac muscle.” said Sonia Albini, PhD, researcher at Genethon, operational manager of the Optidys project.
“Duchenne muscular dystrophy is a devastating disease in which children will inexorably lose their muscle strength. Our ambition is to offer a new generation product with increased efficiency to all patients regardless of the nature of their mutation. Benefiting from this funding is a recognition for Genethon and the scientists who work tirelessly to find life-changing treatments,” said Isabelle Richard, PhD, CNRS Research Director, head of the Progressive Muscular Dystrophies team at Genethon.
This innovative new approach was published on July 13, 2023 in the International Journal of Molecular Sciences.