Muscular Dystrophy Association: Translational Research
Muscular Dystrophy Association (Translational Research)
- LOI: 12/01/2019
- Amount: $300,000 - $1M
- Duration: Up to 2 years
The Muscular Dystrophy Association (MDA)’s translational research program represents an innovative way to fund research that’s designed to move new drug targets into the clinic as rapidly and efficiently as possible. The program was developed in response to the increasing number of promising therapeutic avenues for neuromuscular diseases that have developed from MDA’s discovery research program.
MDA Venture Philanthropy is the Muscular Dystrophy Association’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases. MVP invests in small pharmaceutical and biotech companies and in academics developing treatments and therapies for neuromuscular diseases. MVP funds projects from proof of principle in an animal model through Phase II trials. Projects may include toxicology, manufacturing and scale-up, pre-IND, Phase I or early Phase II trials.
Companies may be public or private, in industry or in academia, and located in the U.S. or internationally. MVP funds academic investigators doing appropriate studies, but encourages corporate collaboration. MVP seeks to apply funding where it will achieve the greatest leverage to increase the number of effective drugs in development for neuromuscular diseases. As such, well-financed projects that will proceed without MVP investment may not be selected for funding.
What kinds of projects are funded?
- Applications should directly relate to a disease in MDA’s program
- High-risk studies at companies that might not otherwise enter the rare neuromuscular disease arena, effectively making potential therapeutics more attractive to investors
- Critical academic, preclinical research to guide whether potential therapeutics developed in academia can be developed as drugs, and to enhance the value of such therapeutics to drug development companies
- Seed funding (< $100K) for promising, early-stage projects, which could lead to further investment by MDA or others
- Research and development projects including target validation, lead identification and optimization, and preclinical efficacy studies
- Preclinical work necessary to file an investigational new drug application (IND), including pharmacokinetic, pharmacodynamic and toxicology studies, as well as compound manufacturing and scale-up
- Phase 1 or early phase 2 clinical trials
- Development of diagnostic, prognostic, and pharmacodynamic biomarkers that may accelerate drug development efforts
- Size and Time Frame of Investment: The award period will be, on average, two years or less, but follow-on investment may be negotiated if all milestones are met and the project moves into later phases of development. The following ranges are guidelines for the value of investment that MVP may put into projects at each stage.
- Research and Development (up to $300,000)
- Preclinical (up to $500,000)
- Early Clinical (Phase 1/2) (up to $1 million)