The main objective of MYOCURE is to develop a novel, innovative and clinically-translatable one-treatment gene therapy platform for patients suffering from rare inherited muscle diseases, specifically focusing on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II.
MYOCURE seeks to overcome the bottlenecks that hamper muscle-directed gene therapy by:
- Boosting gene transfer and expression.
- Minimizing undesirable immune reactions and improving the efficacy and safety.
- Developing a scalable GMP-like manufacturing process.
- Applying for an orphan drug designation for the innovative advanced therapy medicinal product (ATMP).
- Consolidating a Phase I gene therapy clinical trial in patients suffering from MTM and GSD II.