BioPontis Alliance for Rare Diseases Appoints Prestigious Advisory Committee

FOR IMMEDIATE RELEASE
Nov 4, 2016: Raleigh, NC and Brussels, BE

BioPontis Alliance for Rare Diseases Appoints Prestigious Advisory Committee

The non-profit organization, BioPontis Alliance for Rare Diseases (BioPontis), today unveiled the names of the members of its newly formed Scientific Advisory Committee. Developing novel medicines for rare neurological disorders, most of which are genetic in origin, requires innovation in therapeutic approaches. Leading edge knowledge of neuroscience, genomics, clinical neurology, therapeutics development and regulatory sciences must be available to the development team. The Board of Directors sought a panel of experts with in-depth knowledge in all of these areas.

“The selection of our founding Committee members reflects BioPontis’ commitment to greatly increasing the productivity of the bottleneck stages of drug discovery and preclinical development”, states Warren Strittmatter, MD, Co-Chair and Board representative to the BioPontis Scientific Advisory Committee. “Generating more well qualified candidate therapeutic products that are ready to enter clinical trials requires global, broad vision which these international experts bring to the Board of Directors and management at BioPontis. “

The six external and two internal appointees convened their inaugural meeting in September when they considered the focus and approach to transforming the current trickle of therapies being pursued for some of the most devastating and debilitating rare disorders – those affecting the brain and nervous system – into a steady stream.We are very fortunate to have attracted these eminent scientists and physicians, who share our commitment to the mission of accelerating rare disease therapeutics development” says David Spencer, PhD, Chief Scientific Officer and Co-chair of the committee.

We are very fortunate to have attracted these eminent scientists and physicians, who share our commitment to the mission of accelerating rare disease therapeutics development” says David Spencer, PhD, Chief Scientific Officer and Co-chair of the committee.The new Committee appointees are:

The new Committee appointees are:

  • Huda Zoghbi, MD, PhD – Professor in the Departments of Pediatrics, Molecular and Human Genetics, Neurology and Neuroscience at Baylor College of Medicine, Houston, Texas. After her 1999 discovery of the gene responsible for Rett Syndrome, she has been elected to the US National Academy of Sciences, the Institute of Medicine, and the American Association for the Advancement of Science.
  • Jan Gheuens, MD, PhD – Neurologist, currently Deputy Director of Tuberculosis Drug Development at the Bill and Melinda Gates Foundation, Seattle, Washington. Previously at Amgen, where he was Vice President, Global Development Operations.
  • James Lupski, MD – Cullen Professor of Molecular and Human Genetics and Professor of Pediatrics at Baylor College of Medicine, Houston Texas, elected to the Institute of Medicine of the US National Academies of Science and to the American Academy of Arts and Sciences and recently received the 2014 Environmental Mutagenesis and Genomics Society Award.
  • Antonio Federico, MD – Full professor of Neurology and Director Unit Clinical Neurology and Neurometabolic Diseases, Medical School, University of Siena, Siena, Italy Chairman of the Scientific Committee of the European Academy of Neurology.
  • Lynn Fahey McGrath, MD, MPH – Vice-President Regulatory Affairs, RegenxBio, Rockville, MD – She has led regulatory affairs global teams for products from early development through registration and commercialization. Successful registrations of over 50 NMEs/major variations in US, EU (Centralized, MRP, DCP process).
  • Wim Robberecht, MD, PhD – Neurologist and Pharmacologist, Chairman of the Biomedical Sciences and Vice Rector of the University of Leuven, Belgium, and Chairman of the Board of the University Hospitals Leuven. In 2014 he received an award of the Medical Foundation Queen Elisabeth.

About BioPontis Alliance for Rare Diseases
BioPontis Alliance for Rare Diseases is a 501c(3) Public Charity in North Carolina (USA) and a Foundation of Public Utility in Brussels (Belgium). Managed jointly by an international Board and executive team, BioPontis partners with patients’ organizations and academic researchers to bridge the gap from promising science to medicines, ready for uptake by the biopharmaceutical industry. BioPontis Alliance for Rare Diseases is reaching out to all stakeholders in the biopharmaceutical industry, to institutions, charitable funds and foundations, and to individuals who want to directly impact the healthcare outcomes for rare disease patients who live with no hope of treatment today. Contributions to fund and support our programs and mission can be made at http://.biopontisalliance.org or by contacting the contact persons below.

Erik Tambuyzer, PhD, Chairperson
+32 475 615711 (Brussels, Belgium)
erik.tambuyzer@biopontisalliance.org

Barbara Handelin, PhD, CEO
+1 -610- 636-2332 (Philadelphia, PA, USA)
barbara.handelin@biopontisalliance.org