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MVP's fourth portfolio covers all additional neuromuscular diseases in MDA's program, including other muscular dystrophies, Friedreich's ataxia, Charcot-Marie-Tooth disease, myasthenia gravis and various metabolic diseases of muscle. Therapeutic development for many of these diseases is still at an earlier stage than for the major portfolio diseases at this time. When multiple promising projects in a disease area become apparent to MVP staff, additional individual portfolios will be created to focus on specific disease areas.
MVP's current portfolio:
- MVP awarded $731,534 to Repligen Corporation for development of a compound to treat Friedreich's ataxia. MDA's translational research program had previously funded this company for earlier-stage development of this compound, which looks very promising. After successful completion of the MVP project, Repligen anticipates initiating the first trials of the therapy in FA patients.
- MVP awarded Dr. Jerry Mendell at Nationwide Children's Hospital $458,814 to complete the necessary work to start a limb delivery trial of his gene therapy for alpha-sarcoglycan deficient limb-girdle muscular dystrophy. Dr. Mendell already has shown that the therapy works when it is injected into a single site, and hopes that by delivering the therapy to multiple muscles simultaneously this trial will demonstrate increased strength in patients, along with proving that the therapy and delivery method are safe.
- MVP awarded 4s3 Biosciences $260,291 to develop an antibody-mediated method to deliver the gene defective in myotubular myopathy, MTM1, to muscles. This early-stage project will not only offer a potential therapy for myotubular myopathy, but the delivery method may be applicable to other diseases of muscle.
- MVP awarded Matthew Meriggioli at the University of Illinois $530,480 to develop a dosing regime and to complete a small phase 1 trial of GM-CSF in myasthenia gravis. If successful, this study will result in a larger trial that might allow the drug to be marketed for myasthenia gravis.
- MVP awarded Martin Childers at Wake Forest University $369,365 to develop a new treatment for patients with X-linked myotubular myopathy. This project is to test a new gene therapy for myotubular myopathy in animal models prior to moving into human trials. The project is co-funded by the Association France Myopathie (AFM), and is to be completed in collaboration with the French company Généthon.
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