The new collaborative research project MYOCURE aims at advancing the development of new therapies for patients with rare muscle diseases. In particular, MYOCURE will impact on an estimated 20,000 people in the EU suffering from myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II (Pompe’s disease) as well as impact on other muscle disorders. MTM and GSD II are quintessential hereditary muscle diseases for which there is no definitive cure. They are attractive diseases for gene therapy as they comprise a diverse family of rare genetic diseases typically caused by single gene defects, and often provoke significant morbidity and mortality due to skeletal muscle, cardiac and/or diaphragm dysfunction. The costs for treatment and care taking of GSD II patients, amount to €400,000 to €700,000 per year per patient. In the EU, this corresponds to an estimated cost of €4 billion to €7 billion annually. Thus, advancing research in this field is a societal need and a crucial commitment to improve patient care and relieve the healthcare-system.
In this context, MYOCURE was launched to overcome current scientific and societal bottlenecks, as well as to develop aninnovative and clinically applicable gene therapy platform to cure rare hereditary muscle disorders. Equipped with a budget of about €6 million, the novel approaches that will be developed in MYOCURE shall form the basis for a one-time treatment, which is expected to provide enhanced patient treatment and significantly reduce healthcare costs.
Professor Marinee Chuah from the Vrije Universiteit Brussels and Coordinator of the project stresses the importance of the social and economic impact of the project: “Establishing such a clinically translatable one-time treatment platform will contribute significantly to a higher patients” quality of life. Moreover, the social impact stretches out to the families and relatives of the affected persons, as the need for home or hospital care is decreased, hence reducing the overall social and economic costs?. She further emphasizes that one of the unique strengths of MYOCURE is the combination of most recent innovations in gene therapy with the latest insights in immune control to maximize efficacy and safety. Furthermore, MYOCURE will enhance expertise in the field of orphan and rare diseases, as well as strengthen the competitiveness and growth of companies in the EU by developing innovations meeting the needs of European and global markets.
We participate in MYOCURE project as a regulatory partner leading the following activities:
– Scientific Advice with the EMA for the treatment of MTM and GSD II.
– Orphan Drug Designation Applications for both indications, MTM and GSD II.
– Consolidation of a clinical development plan to be agreed with the EMA CHMP in a Follow-Up Scientific Advice.
MYOCURE brings together leading experts in science and industry from eight European partner institutions to reach these goals and successfully implement the project’s ambitious work plan.
We offer comprehensive services for Drug Development and Regulatory Affairs to Pharma, Biotech and Medical Devices companies.
Since 2000, we have consistently grown and now have over 70 professional consultants with backgrounds in Life sciences (Pharmacy, Chemistry, Biology, Biochemistry, Biotechnology, Medicine and Veterinary Medicine).
Our consultants are experts in their field and are in direct contact with EU agencies (EMA and NCAs) for the development and implementation of new regulatory standards.
MYOCURE: This project has received funding from the
European Union?s Horizon 2020 research and innovation programme under grant
agreement No
667751.
