First project on Charcot-Marie-Tooth Disease


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 invalidating neuropathy.

Image: Image shows diagnostic form, on which Charcot-Marie-Tooth disease is written and tablets packs and stethoscope are lying; Copyright:

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.; Source: Flanders Interuniversity Institute for Biotechnology (VIB)

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