BioPontis Alliance, HNF Form Partnership To Develop Rare Disease Treatment
By C. Rajan, contributing writer
North Carolina-based non-profit organization, BioPontis Alliance for Rare Disease, has formed a partnership with New York based philanthropy, Hereditary Neuropathy Foundation (HNF), to develop drug candidates for the treatment of rare disease Charcot- Marie-Tooth (CMT) Disease.
The recently founded philanthropic organization, BioPontis Alliance, was designed exclusively to translate discovery science into new therapies for rare diseases by forming collaborative partnerships with patient organizations, research scientists, and pharmaceutical companies.
This joint venture with HNF is BioPontis Alliance’s first collaboration with a patient organization, and aims to bring together researchers and all CMT-dedicated organizations in order to find a cure for CMT.
BioPontis Alliance will be responsible for managing the joint venture’s scientific execution, regulatory strategy, patenting, and business partnering, while HNF will manage patient-related activities such as patient registries, clinical outcome measure research, and advocacy to support the patient community. HNF will also continue to support other translational research efforts until they are ready to play a larger role in the collaboration.
"This is an open platform, that can incorporate multiple approaches to solving the puzzle of how to save the nerve tissues that are under siege in CMT," says Richard Basile, co-founder and COO at BioPontis Alliance. "We have designed our model for conducting drug discovery with the expectation that international collaboration among scientists, patient organizations, biopharmaceutical companies, and philanthropic supporters is essential."
While there are many research projects being performed on various rare diseases and new government initiatives to advance therapies for rare diseases, most of these conditions still lack treatment options and remain a huge unmet medical need.
According to the company’s press release, there are over 350 million people worldwide who suffer from rare diseases collectively, including a large number of children. There are currently 7,000 rare diseases in existence, of which 95 percent have no treatment.
Among the known rare diseases, CMT is the most common inherited neuropathy. It is a gradually progressive disease, which causes the legs and arms to become deformed. Chronic, severe pain is common in CMT and most patients lose mobility over time. The disease affects 1 in 2,500 people, with over 2.6 million patients worldwide. Since the discovery of the disease, scientists have discovered most of the genes associated with the disease, however there have been no treatments developed for CMT yet.