Christopher Austin, Founding Director of the National Center for Advancing Translational Sciences, to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention

ATLANTA (July 12, 2022) Christopher Austin, M.D., CEO of Vesalius Therapeutics, CEO-Partner of Flagship Pioneering and Founding Director of the National Center for Advancing Translational Sciences (NCATS) will be the keynote speaker on the first day of the second annual Global CMT Research Convention, it was announced today by Cleary Simpson, CMT Research Foundation’s CEO. The convention, to be held in Cambridge, MA, September 16th-17th, is a weekend centered on identifying treatments for all types of CMT, where patients, researchers, biotech and pharma companies all come together to catalyze progress. Last year, 160 researchers from 20 different countries and 300 patients from 34 different counties helped launch the first Global CMT Convention.

At this year’s convention, the CMT Research Foundation will salute the achievements and pioneers that have paved the way towards a cure and preview the work to come for CMT treatments. Attendees both live and virtual will be introduced to the Boston-centric world of biotechnology and explore how it will influence the future of CMT drug development. Among the other speakers during the two-day event will be:

  • Dr. Vincent Timmerman, who found the CMT1A gene in 1991
  • Dr. James Lupski, who also found the CMT1A gene in 1991 in the same month as Dr. Timmerman
  • Dr. Reza Seyedsadjadi, who leads the CMT clinic at Harvard/Massachusetts General Hospital. He will also bring several of his CMT clinical colleagues with him
  • Dr. Paul August, Chief Scientific Officer at ReviR
  • Dr. Arthur Suckow, the CEO of DTx and Dr. Scott Harper, a professor at Nationwide Children’s Hospital, who are both leading CMT drug development programs
  • Dr. Maurizio D’Antonio, who discovered the role of the unfolded protein response in CMT
  • Dr. Rudolph Martini, a leader in the study of the immune system in demyelinating forms of the disease
  • Dr. Rachel Bailey, who is developing gene therapies for CMT

Dr. Austin is a trained clinician and geneticist, with more than 20 years of experience in translational research in both the public and private sectors. He joined Flagship Pioneering, a bioplatform innovation company focused on human health and sustainability, as CEO-Partner in 2021 and serves as Chief Executive Officer of Vesalius Therapeutics. Before that, he was the founding Director of NCATS at the National Institutes of Health (NIH) where he transformed translation – the process by which interventions that benefit patients are developed and deployed – from an empirical process into a predictive science.

“Friday September 16th is a day for researchers to share the latest in CMT drug development, establish new connections and collaborations and discuss pathways to treatments,” says Ms. Simpson. “Saturday September 17th is a day for patients and their families. We will give special attention to clinical trials; how they operate, why to participate in them, and when to expect them. It is important for researchers and patients to interact directly, and we are pleased to provide that forum.”

“Once again academic researchers, industry partners, regulatory agencies, and patients are uniting to attack key problems in drug development for CMT,” says Keith N. Fargo, PhD and CMTRF’s Chief Scientific Officer “This is truly an extraordinary opportunity to network with the people most critical to finding a cure for the 3 million people around the world with CMT.”

Among the sponsors helping to underwrite the event are: DTx, and Armatus Bio. A complete calendar of events can be found here.

The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research for drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.

*Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in deterioration of peripheral nerves that control sensory information and muscle function of the foot/lower leg and hand/forearm leading to significant problems with movement, touch, and balance as it advances. CMT can vary greatly in severity, even within the same family. CMT can cause severe disability and in rare instances, even death. At the moment, there is no treatment or cure for CMT.