PPMD Venture Pathways

PPMD Venture Pathways is the venture philanthropy program of PPMD, providing industry investment to accelerate therapeutic development.

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.

Through PPMD Venture Pathways, our investment enables companies to complete critical studies needed to secure significant funding required to advance investigational products to the clinic.

The goal of these investments is to increase PPMD’s return on mission by moving potential therapies into the clinic. If these investments result in any financial gains, those funds will be reinvested into our research strategy, supporting the continued advancement of therapies. The goal of this program, like PPMD’s other research strategies, is to get effective and safe therapies to the clinic and then into the hands of our community as quickly as possible.

Pathways for Impact

For the past 30 years, PPMD has funded more than $55 million in research to enhance our knowledge of Duchenne and Becker muscular dystrophy. 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 space today.

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.

Advancing Pathways Forward

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

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 the development of IND packages, and push therapies into clinical trials.

To learn more about how you can support PPMD Venture Pathways please reach out to kaylan@parentprojectmd.org

For Families

Our primary focus is on supporting research that we believe will provide benefit to those living with dystrophinopathies and which have a realistic probability of being translated to patients. While we hope that a return can be realized to spur therapeutic development for Duchenne/Becker, our guiding principle remains on the scientific merit of the approaches. See below for investments through the Venture Philanthropy Program to date:

Myosana Therapeutics

We invested $850,000 towards developing a non-viral platform for systemic delivery of full-length dystrophin.
- Company has completed a $5 million seed round with an angel investor and Duchenne non-profit

Satellos Biosciences

We invested $1,000,000 to the development of a small molecule to restore asymmetric division of stem cells in the absence of dystrophin and restore the regenerative capacity of muscle
Company is now publicly traded company on the Toronto Venture Stock Exchange
- Raised $55 million in public offering
- Plans to initiate IND by mid-2024

ReveraGen BioPharma

We invested $750,000 into the development of VBP15 (Vamorolone), a dissociated steroid for use in patients with dystrophinopathies
Vamorolone has been licensed by Santhera Pharmaceuticals and Catalyst Pharmaceuticals (Catalyst has commercialization rights for U.S., Canada, and Mexico)
On October 26, 2023 the FDA approved AGAMREE (vamorolone) for individuals diagnosed with Duchenne from age 2 years and older.

Kinea Bio

PPMD provided to the development of a next-generation adeno-associated virus (AAV) mediated midi-dystrophin gene replacement therapy
Funding will enable Kinea to complete critical studies needed to secure funding required to advance their investigational product to the clinic

For Researchers

Application process for grant funding through PPMD’s Venture Philanthropy Program

PPMD is looking to invest in early to mid-stage biotech companies seeking support to advance their discovery, pre-clinical, or clinical development. All companies will need to participate in a due-diligence review covering their product, development plans, scientific and business team members, strategies for future investment, and projected timeline to the clinic. If you are interested in initiating discussions with PPMD’s Venture Pathways Advisory Committee please reach out to PPMDs VP of Research and Clinical Innovation, Eric@parentprojectmd.org.

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