For the past 30 years PPMD has funded more than $55 million in research to enhance our knowledge of dystrophinopathies. We’ve provided funding for a variety of areas of research from basic research to preclinical work and natural history studies laying the foundation for the tremendous  therapeutic development we see in the Duchenne space today.

Part of PPMD’s core mission is to accelerate research which includes identifying promising therapeutic strategies and spurring their development and translation into the clinic with hope for approval and access for patients.

In this ever-evolving landscape of drug development, PPMD has focused our approach to supporting translational therapies as a part of our overarching research strategy. Recently, we have watched as more of our academic partners and thought leaders look for ways to expedite their research programs, moving them from bench to bedside. The challenge for many of these groups is  generating sufficient data packages to entice significant investors, to make the sizable investments required to take a product through to a human clinical trial.  This includes all of the safety, efficacy, and regulatory steps needed.

Recognizing this challenge and identifying a gap that PPMD can help fill, we have begun investing in early-stage biopharmaceutical companies where we can de-risk and catalyze development of novel therapies to treat dystrophinopathies.

This type of early-stage industry investment (often referred to as venture philanthropy) enables companies to complete critical studies needed to secure significant funding required to advance investigational products to the clinic. These investments also create the opportunity for PPMD to have a financial return on the investments which we will use to reinvest into our research strategy and support additional work in the future.

Our interest is not focused on our return on investment, but our return on mission. The goal of this program, like PPMD’s other research strategies, is to get effective and safe therapies to the clinic then into the hands of our community as quickly as possible. We are excited to add this approach to our historically successful research strategy., which we will continue to use to drive our support of critical basic science and natural history studies. Through this process we will measure our success by how our investments drive innovative science, disseminate learnings through publication, contribute to development of IND packages, and push therapies into clinical trials.


To learn more about how you can support PPMD’s Venture Philanthropy Program, please reach out to