Treating Rare Hereditary Muscle Disorders: Interviews with Partners of H2020 Project MYOCURE
Now published: The EU H2020 project MYOCURE has released an interview series produced during the 25th Anniversary Congress of the European Society of Gene & Cell Therapy (ESGCT) in Berlin. As dissemination partner, Eurice took care of the complete production process of the interviews.
The series illustrates the objectives and activities of the multi-national, interdisciplinary consortium working together in this four-year research initiative. The partners highlight their personal motivation and the different expertise they bring into the project while addressing some key challenges that need to be overcome towards the ultimate goal of bringing the new technologies to the clinic and to the patients. MYOCURE was launched in January 2016 with the aim of developing a novel, 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. MTM and GSD II are severe, life-threatening diseases for which there is no definitive cure yet. Since this disease group comprises a number of rare genetic diseases typically caused by single gene defects, MYOCURE will have an impact beyond MTM and GSD II and the innovative platform technology will concern most rare genetic diseases affecting the muscle. MYOCURE will have a favourable impact on the quality of life of patients and their families and will reduce the societal costs associated with their treatment and care. Furthermore, the project will improve the medical knowledge and competitiveness of Europe in the field of gene therapy technologies.
Two years into the project, MYOCURE has already demonstrated the innovation potential generating a vector which outperforms the current state of the art. The next steps are to improve the manufacturing of the new therapies and to prepare for the ultimate Orphan Drug Designation application.