Asphalion participated in the 2nd Annual Meeting of the H2020 project MYOCURE, the 19th and 20th of March at the University Pierre and Marie Curie (Sorbonne Université) – Campus des Cordeliers, in Paris.
The Annual meetings takes place once a year and include representatives from each partner of the MYOCURE consortium. The most recent data from all partners was presented and open discussions and constructive interactions occurred and empowered us to move forward the project with fruitfully ideas.
MYOCURE aims at developing new therapeutic products based on gene therapy for patients suffering from rare inherited muscle diseases. The research specifically focuses on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II. Both diseases are severe, potentially life-threatening and current treatment is suboptimal. You can find more information at Myocure website (http://myocure.eu/).
Asphalion participates in the MYOCURE project as a regulatory partner addressing the regulatory issues related to the clinical development of the MYOCURE innovative gene therapy platform to ultimately treat patients suffering from GSDII or MTM. In particular, Asphalion develops and conducts the following activities:
- Early Scientific Advice given by EMA for the treatment of MTM and GSDII
- Orphan Drug Designation (ODD) for prototype AAV vector for the treatment of MTM
- Orphan Drug Designation (ODD) for prototype AAV vector for the treatment of GSD II
- Late Scientific Advice given by EMA including the design of clinical trial based on the prototype AAV vectors for the treatment of MTM and GSDII
The MYOCURE project brings together the expertise of a number of European groups (Spain, Belgium, France, UK, and Germany) in a number of fields required for an adequate development of the gene therapy project.
MYOCURE: This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667751.
