BioPontis Alliance for Rare Disease Offers Enabling Bridge Between Basic Research and Treatments

For Immediate Release

BioPontis Alliance for Rare Disease Offers Enabling Bridge Between Basic Research and Treatments


Raleigh, NC – October 6, 2014. A team of seasoned drug development professionals, patient advocacy leaders and regulatory professionals today announced the founding of BioPontis Alliance for Rare Disease.  BioPontis Alliance is an international philanthropic organization, bringing a professional drug discovery and development engine to help fill the therapy void for rare diseases, 95% of which have no treatment. Although individually rare, there are 7000 such diseases, such that, collectively, more people suffer from rare diseases than from AIDS and cancer combined.  Most of the 350 million affected worldwide are children.

BioPontis Alliance for Rare Disease is the first non-profit organization designed exclusively to translate discovery science into candidates for new therapies for rare diseases.  The mission is to be an ally to rare disease patient organizations and academic scientists to translate research into potential treatments. BioPontis will be announcing their first patient organization joint venture later this month.

While patient organizations continue to fund innovative research, ideas for treatments often become stalled at the doorway of drug development. “We applaud recent successes achieved by patient organizations and academic centers driving drug discovery for some of the many rare diseases. However, we believe that it is not pragmatic to expect each rare patient organization to organize the complex wheelhouse of resources and skills needed to translate basic research into medicines on their own” says Barbara Handelin, PhD, cofounder and CEO at BioPontis Alliance.

The BioPontis Alliance team has this expertise and plans to bridge the gap by partnering worldwide with patient organizations, research scientists and pharmaceutical companies through the formation of joint venture structures designed to advance ideas for treatments into drug candidates. “Research that never gets off the lab bench and tried in patients can never be a cure,” says Marlene Haffner MD, MPH, BioPontis Alliance Vice Chairwoman, and past head of the FDA Office of Orphan Products Development. “We believe strongly that if those who are skilled in the science and business of developing drugs can align to the academic, philanthropic and patient driven passion to create new treatments, we make real progress on bringing that 95% down significantly” joins BioPontis’ Board Chairman, Erik Tambuyzer, PhD also Chair at the Center for Medical Innovation, (Leuven, Belgium) and former senior executive at Genzyme. “BioPontis will derisk the early drug development and look for licensees to advance into later stage clinical trials”, he maintains.

The philanthropic organization has international perspective and has attracted a powerful Board of Directors and advisors. Drs. Tambuyzer and Haffner are joined by Susan Kahn, Executive Director, National Tay- Sachs and Allied Diseases Association; Warren Strittmatter, MD, former Chief of Neurology at Duke University; and Pat Stocker PhD, Executive Education Consultant for the Office of Executive Programs (OEP) at the Robert H. Smith School of Business at the University of Maryland where she previously served as Associate Dean.

About BioPontis Alliance for Rare Disease

BioPontis Alliance for Rare Disease is a North Carolina non-profit corporation that partners with patient organizations and academic researchers to carry out the earliest stages of product development to bridge the gap between ideas for treatments and drug compounds that are ready for uptake by the biopharmaceutical and biotech industry. BioPontis Alliance for Rare Disease is being established as a global non-profit development hub, inviting corporate, private and foundation philanthropic support.


Barbara Handelin, PhD, CEO

In  Europe:
Erik Tambuyzer,PhD, Chairman
+32 475 61 57 11