The EU H2020
has released an interview series produced during the 25th Anniversary Congress of the European Society of Gene & Cell Therapy (ESGCT
) in Berlin. 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.
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
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
brings together a multidisciplinary project consortium with outstanding experience in the fields of gene therapy, regulatory issues and EU project management. The project is funded by the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667751.
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.
experts attended the 25th Anniversary Congress of the European Society of Gene & Cell Therapy (ESGCT) in Berlin in October 2018. Joel Montané
, Scientific & Regulatory Affairs Officer at Asphalion
, participated in the interview series to explain his experience at MYOCURE
project. Watch the interview:
MYOCURE: This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667751.