Last week Paul August and I attended the BIO International Convention, a conference with over 16,000 leaders from the biotech and pharmaceutical industry. We met a number of companies interested in advancing drug development for CMT. There are four primary factors which make CMT attractive to them from a business perspective:

  • There is a high unmet need; there are over 2.8 million people worldwide living with CMT without treatments.
  • Because CMT is categorized as a rare disease by the FDA, biotech and pharma are incentivized to develop therapeutic approaches to CMT.
  • CMT is a genetic disease and with the recent approvals of gene therapy for other diseases, using this approach to treat CMT is rational and viable.
  • With the current body of knowledge and advances in technology, the landscape for drug development has never been better for CMT.

Additionally, courtesy of the leaders at BIO,I had the opportunity to speak to a group of leaders in the industry in a public forum, building a case for support for CMT research and working with the CMT Research Foundation.  Through these meetings and presentations, Paul and I built momentum within the biotech and pharma industry, many of whom expressed a strong interest in CMT.  We will continue to advance the conversations to find the most promising and urgent partnerships to develop treatments for CMT.