The Hereditary Neuropathy Foundation and BioPontis Alliance for Rare Disease Partner to Develop Treatments for Charcot-Marie-Tooth

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Hereditary Neuropathy Foundation; BioPontis Alliance for Rare Disease

Oct 20, 2014, 08:02 ET

NEW YORK and RALEIGH, N.C., Oct. 20, 2014 /PRNewswire/ -- BioPontis Alliance for Rare Disease and the Hereditary Neuropathy Foundation (HNF), both philanthropies, announced today the creation of a joint venture to develop drug candidates for the treatment of the rare disease known as Charcot- Marie-Tooth (CMT) Disease.

BioPontis Alliance announced its alliance model earlier this month. Today's partnership announcement with HNF is a first demonstration of a collaborative model where researchers and all CMT dedicated organizations can come together for a common cause – treatments.

CMT is the most common inherited neuropathy, affecting 1 in 2500 people, some 2.6 million worldwide. It is a progressive disease, often beginning so subtly that it may go undiagnosed for years. As the disease progresses, legs and arms become deformed and difficult to use. Those affected by CMT often lose the ability to walk and may become confined to a wheelchair. Chronic, severe pain is common. There is no treatment for CMT which was first identified more than 100 years ago by Drs. Charcot, Marie, and Tooth. Fortunately, in the past 20 years, scientists have discovered most of the genes associated with the disease.

Patient organizations like HNF and federal grants from National Institutes of Health have supported basic research into the cause of CMT and early translational research to develop critical tools for drug development, with the goal that research would lead quickly to treatment. Progress can become stalled because drug development requires a complex array of more engineering like technical capabilities, capabilities not available to patients and academic researchers alike. Also new attention must be paid to patenting and aligning to the interest/requirements of the drug industry players who will be needed to bring treatments to patients.

"Although there is growing pharmaceutical industry and private investment activity around rare diseases," comments Allison Moore, founder and CEO at HNF, "we have experienced that they need to focus their investments primarily in drug candidates that are ready to enter clinical trials" – not at the earlier stage where potential treatments are first being developed and tested in cells or animal models. "This is why a strategic partnership with BioPontis Alliance makes sense for us - they can provide the scientific, business and patenting structure to help bridge to those pharmaceutical companies," says Carol Liu, Co-Chair of the Board at HNF.

In the JV, BioPontis Alliance will manage the project's scientific execution, regulatory strategy, patenting and business partnering. HNF will manage resources such as patient registries, bio-banking where appropriate, and will continue to support other translational research until ready to enter the JV. With the drug development effort organized into this structure, HNF can focus on critical adjunct initiatives, such as patient communities to conduct market research, clinical patient registries, clinical outcome measure research and advocacy to support the patient community.

"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."

About Hereditary Neuropathy Foundation

HNF, a non-profit 501(c) 3 organization whose mission is to increase awareness and accurate diagnosis of CMT and related inherited neuropathies, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures. HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for CMT. Currently TRIAD involves many groups that span the drug discovery, drug development, and diagnostics continuum.

Media Contact  Allison Moore, CEO  HNF  212-722-8396  [email protected]

About BIoPontis Alliance for Rare Disease

BioPontis Alliance for Rare Disease is a North Carolina non-profit corporation dedicated to developing new therapeutic agents to treat rare diseases. The niche philanthropy partners with patient organizations, academic researchers and biopharmaceutical companies to bridge the gap between scientific discovery and drug development. BioPontis Alliance for Rare Disease is a global non-profit development hub, inviting corporate, private and foundation philanthropic support. www.biopontisalliance.org