HAMILTON, N.J. and STATEN ISLAND, N.Y., Aug. 21, 2018 /PRNewswire-iReach/ -- Several gene therapies for Duchenne muscular dystrophy have advanced to clinical trials in recent months, offering great hope to the patient community. However, progress has been mired by manufacturing and safety setbacks, which resulted in clinical holds for two of the companies. Sadly, older patients are excluded from the initial series of gene therapy clinical trials, in part because they generally have more advanced disease which may trigger a more rigorous immune reaction to the viral capsid, the therapeutic gene, or manufacturing contaminants.
This request for proposals will identify top projects that aim to overcome gene therapy safety and manufacturing challenges that are particularly problematic for older patients but also exist in the younger patient population. The foundations are also interested in supporting clinical trials outside of gene therapies that include older patients.
Scientists and clinicians are encouraged to reach out to Robert Capolongo and David Schultz using the two emails below to initiate the application process. Applicants will be required to submit a one-page letter-of-intent and selected projects will be invited to submit a full proposal. Funding is currently available and inquiries are encouraged immediately, although there is no formal deadline. Full details will be provided by email.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a neuromuscular disorder that occurs in approximately 1:3600 males and some females which leads to iterative cycles of muscle degeneration and regeneration resulting in inflammation, fibrosis, progressive decline of motor, respiratory, and cardiac function, and premature death. To date, there are no satisfactory therapies and Duchenne remains a severe unmet need.
About the Foundations
Michael's Cause was founded in 2011 after their son Michael was diagnosed with Duchenne Muscular Dystrophy. Ryan's Quest was founded in 2008 after their son Ryan was diagnosed. The Foundations raise awareness of Duchenne and direct all funding to the best medical and scientific research to find better treatments and a future cure for this disease. The Foundations continue to collaborate with Pietro's Fight, Powers Promise, and many others to support this and other drug development projects. To date, Michael's Cause and Ryan's Quest have directed over $3.9 million dollars towards research.
Contact Information for RFP and Grant Inquiries
President, Michael's Cause
President, Ryan's Quest