Rare Disease Report: New Partnership To Encourage Drug Discovery for Charcot-Marie-Tooth Disease

October 30, 2014

BioPontis Alliance for Rare Disease and the Hereditary Neuropathy Foundation have created a joint venture to develop drug candidates for the treatment of Charcot-Marie-Tooth disease.

Charcot-Marie-Tooth neuropathy is an inherited disorder affecting the peripheral motor and sensory neurons. Although it is an inherited condition, the main signs and symptoms such as decreased muscle size and weakness may not present until adolescence of early adulthood.

Signs and symptoms of Charcot-Marie-Tooth disease vary greatly but may include:

  • Weakness in your legs, ankles and feet
  • Loss of muscle bulk in legs and feet
  • High foot arches
  • Curled toes
  • Decreased ability to run
  • Difficulty lifting your foot at the ankle
  • Awkward or higher than normal step
  • Frequent tripping or falling
  • Decreased sensation or a loss of feeling in your legs and feet

At present, there is no cure or treatment for Charcot- Marie-Tooth disease but Biopontis and the HNF want to change that. And they believe it starts at the preclinical level.

“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 joint venture, BioPontis Alliance will manage the project’s scientific execution, regulatory strategy, patenting and business partnering while HNF will manage resources such as patient registries, bio-banking, etc.

Biopontis Alliance for Rare Diseases

The newly formed non-profit corporation Biopontis is designed to develop niche philanthropy partners with patient organizations, academic researchers and biopharmaceutical companies to bridge the gap between scientific discovery and drug development. Bioponitis is a who’s who of the rare disease community including board of directors with industry, regulatory, patient advocacy, investment, and clinical backgrounds (Erik Tambuyzer, Marlene Haffner, Susan Kahn, Warren Strittmatter, Pat Stocker).

The non-profit corporation is led by CEO Barbara Handelin and COO Rickard Basile, and it is specifically designed to bring together people wanting to conduct preclinical studies (scientists, biotechs, etc), people wanting to invest in those studies (venture capitalists, philanthropists), and people who need those studies (patient advocacy groups).  The joint venture with NPF is the first of many partnerships with patient advocacy groups the non-profit corporation hopes to develop.

COO Richard Basile said, “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.”

Hereditary Neuropathy Foundation

HNF, a non-profit 501(c) 3 organization whose mission is to increase awareness and accurate diagnosis of Charcot- Marie-Tooth disease 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.

Recently, HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for Charcot- Marie-Tooth disease. Currently TRIAD involves many groups that span the drug discovery, drug development, and diagnostics continuum.

Image couresy Biopontis Alliance for Rare Diseases

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