Allison Moore, HNF CEO, and Joy Aldrich, HNF Advocacy Director, represented HNF as sponsors at this important conference, which was held in Chateau Mont Sainte Anne, Canada, from June 27 – July 2. Over 500 scientists, clinicians and CMT industry leaders that work specifically on nerve diseases presented their current research projects. The last three days, specifically dedicated to CMT research, were filled with over 150 presentations and posters.
At the conference, HNF connected with their TRIAD funded researchers and were encouraged by the progress of our CMT2 research program. Andrew Grierson, PhD, Sheffield Institute of Translational Neuroscience, presented his work on the zebrafish preclinical model for CMT2A. Rachel Bailey, a postdoctoral research associate – UNC Gene Therapy Center, made a featured presentation, “Development of a Gene Therapy for Giant Axonal Neuropathy (GAN)”. We were also able to connect with renown scientists such as Ludo Van Den Bosch and Veronick Benoy, KU Leuven & VIB, Belgium, who are studying the therapeutic potential of HDAC6 inhibitors in different forms of CMT2. These are important contacts as we continue the drug discovery path towards treatments for CMT2.
A conference highlight was the presentation of Michael Sereda’s MD, Ph.D and Thomas Prukop, MD from the Max Planck Institute (Göttingen, Germany). They showed promising results obtained in their CMT 1A young rat model treated with Pharnext’s PXT-3003. This might lead to the initiation of this drug candidate development in children affected with CMT 1A.
As sponsors of the conference, HNF had an information table where we were able to bring more attention to our TRIAD research programs, which emphasize collaboration and support of clinical trials. In addition, we also presented a poster with data on our clinical patient registry, GRIN. Our sponsorship not only increased awareness of our patient advocacy foundation, it opened up discussions with world-class researchers on new approaches to unlocking additional pathways to gene therapy to cure some of the rarer forms of CMT. Also, highlighted was HNF’s collaboration with Jackson Labs and the newest endeavor to support a repository of mice with all forms of CMT to the research community. This is very important to scientists that are studying and understanding disease as well as experiments to validate clinical compounds for drug discovery.
It was an incredibly successful and informative week. There is an amazing amount of work being done around the world to find treatments for CMT and related inherited neuropathies!