BioPontis Alliance for Rare Diseases and VIB announce strategic partnership on rare diseases – First project on Charcot-Marie-Tooth Disease.

FOR RELEASE: September 2nd, 2016

BioPontis Alliance for Rare Diseases and VIB announce strategic partnership on rare diseases – First project on Charcot-Marie-Tooth Disease.

Ghent & Brussels, Belgium and Raleigh, NC, USA.

Today, BioPontis Alliance for Rare Diseases, a unique international nonprofit organization, and VIB, an excellence-based Life Science Research Institute in Belgium, announced a strategic partnership in rare diseases. The first program is aimed at developing a treatment for one type of Charcot Marie Tooth disease (CMT), a rare, progressive and debilitating neuropathy. There are no therapies available for CMT patients today, although it affects 1/2500 worldwide.

The partnership will advance the work of one of VIB’s premier researchers, Dr. Albena Jordanova at VIB-University of Antwerp. Patients with the form of CMT under investigation present symptoms common for many neuropathies and other neuromuscular diseases such as gradual weakness and wasting of the limbs, sensory loss and inability to do daily tasks, but have an earlier onset of the disease. Lessons learned from the research into this specific form of CMT may hopefully help develop treatments for other forms of the disease.

The partnership blends the basic research and early stage translation resources at VIB with the professional therapeutics development expertise in the rare disease field at BioPontis Alliance. The public service missions of both partners enable the combined team to take bold steps in cracking the complex therapeutic challenges that a disease like CMT presents. By joining forces both partners can step in earlier than most for-profit developers could justify.

VIB and BioPontis Alliance will also collaborate to identify and jointly develop other VIB research projects with high promise to be developed into treatments. VIB brings a track record in translating promising basic research into valuable innovations and transition them into further development. Recently, an industry-trained drug discovery team, VIB Discovery Sciences, was installed hereto. BioPontis Alliance brings its innovative partnerships approach working with rare disease patients’ organizations and its scientific, regulatory and biopharmaceutical industry expertise.

“We as VIB scientists are committed to not only unravel the basic mechanisms underlying major societal challenges. This partnership gives a unique opportunity for us, to accomplish one of the major long term aims of our research, namely translating basic findings into treatment opportunities for patients”, commented Albena Jordanova, Principal Investigator at VIB.

“With the start of our VIB Discovery Sciences team, we support our scientists and partners to engage in this work. This alliance with Biopontis is a nice example of how to make this work in rare neurodegenerative diseases with huge unmet needs”, added Jérôme Van Biervliet, Sr Business Development Manager at VIB.

“We at BioPontis Alliance are very excited to be starting our first discovery project within 6 months of being operational. We find great synergy with the mission of VIB and look forward to work with them on this first exciting project and to the expansion of our partnership into future projects in the field of rare neurological diseases. We aim to identify and develop a portfolio of new candidate medicines for transfer to the biopharmaceutical industry where clinical trials, manufacturing and distribution to patients is accomplished”, said Barbara Handelin CEO at BioPontis Alliance.

Rare diseases taken together represent one of the largest underserved patient populations worldwide, more than all the HIV/AIDS patients and cancer patients combined. The genetic cause of 4000 of the 7-8000 rare diseases are known, but the translation of that knowledge into therapies remains disorganized and vastly underfunded. This results in less than 5% of rare disease patients having any treatment options whatsoever.

About BioPontis Alliance for Rare Disease

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 or by contacting the contact persons below.

About VIB

VIB, with multiple sites in Flanders, conducts basic research in life sciences. VIB researchers push the boundaries of what is known about molecular mechanisms and how they rule living organisms such as human beings, animals, plants and microorganisms. These breakthroughs are translated to tangible innovations for the benefit of society through tech transfer. Based on a close partnership with the 5 Flemish universities in Belgium and supported by a solid funding program, VIB unites 75 research groups focusing on basic life science research in one single institute. Through partnering with private partners or establishing new start-ups, VIB’s technology transfer translates research results into economic ventures which, in time, lead to new products that can be used in medicine, agriculture and other applications. VIB also engages actively in the public debate on biotechnology by developing and disseminating a wide range of science-based information about all aspects of life science research.



BioPontis Alliance for Rare Diseases:
Erik Tambuyzer, Chair of the Board
+32 475 615711 (Brussels, Belgium)

Barbara Handelin, CEO
+1 -610- 651-7847 (Philadelphia, PA, USA)

Johan Cardoen, Managing Director
+32 475 98 45 25

Jérôme Van Biervliet, Sr Business Development Manager and Manager VIB Discovery Sciences
+32 473 92 88 50