This project focuses on developing an innovative gene therapy approach for neuromuscular disorders, particularly X-linked Myotubular Myopathy (MTM1) and Duchenne Muscular Dystrophy (DMD). Current gene therapies using recombinant adeno-associated virus (rAAV) vectors face challenges such as high systemic doses, off-target effects, and pre-existing neutralizing antibodies.

The proposed solution involves the chemical modification of rAAV capsids through bioconjugation techniques to improve muscle tissue targeting while reducing non-specific diffusion to other organs. By employing novel conjugation strategies, the project aims to enhance therapeutic efficiency and safety.

The initiative is a collaboration between Nantes University’s CEISAM and TaRGeT laboratories, alongside Coave Therapeutics, leveraging expertise in organic chemistry, bioconjugation, and gene therapy. The expected outcome is the development of optimized chemically modified rAAV vectors with improved specificity, enabling safer and more effective gene therapies for neuromuscular diseases.

David DENIAUD

Project leader

TaRGeT

UMR INSERM 1089