On June 8 & 9, 2018, scientific and medical researchers from academia and industry convened for the FSH Society’s 23rd annual International Research Conference (IRC) in Las Vegas, NV. Biocells was proud to support this meeting as well as the patient focused FSH Connect meeting. The FSH IRC meeting attracted attendees to present and discuss new developments in FSHD research, reinforce collaborative efforts, and facilitate new initiatives. We were pleased to have Dr. Uli Schmidt, President and Managing Director of Genea Biocells, present data on Genea’s FSHD targeted therapeutic, GBC0905, which was recently granted Orphan Drug Designation by the FDA.
The first part of the conference was primarily basic research focused, covering the latest updates on molecular modulators of FSHD, the regulation and mechanisms of DUX4, and state of the art FSHD animal models. The second part of the conference focused on accurate clinical diagnosis of FSHD patients and measurement of potential clinical outcomes including MRI, ultrasound, and electrical myography impedance. The final session highlighted therapeutic developments for the treatment of FSHD by both industry and academic researchers. Clinical data was presented for Acceleron’s ACE-083 treatment in FSHD patients by Dr. Jeff Statland. Both small molecule and gene therapy approaches were described by industry and academic researchers for the treatment of FSHD. The session was closed out by Dr. Schmidt who spoke about Biocells’ targeted therapeutic candidate GBC0905 which potently suppresses DUX4 function, prevents the activation of DUX4 target genes, and protects affected skeletal muscle cells in a dose-dependent fashion and, importantly, without affecting normal myogenesis. All the talks demonstrated the tremendous amount of progress that is being made to understand the underpinnings of FSHD and the development of efficacious treatments for FSHD patients.
Following the FSH IRC meeting, the FSHD Connect Conference on June 9-10, 2018 brought together hundreds of patients, family members, researchers, physicians and health experts for a day-and-a-half of immersive learning and community building was in Las Vegas, NV. As part of the “Road to Treatments” session, Biocells was joined by both academic and industry researchers leading the charge to develop therapeutics for FSHD patients. Dr. Monica Hayhurst Bennett, Director of Preclinical Development at Genea Biocells, presented progress on Biocell’s GBC0905 for the treatment of FSHD as part of a panel discussion directly addressing FSHD patients and their families.