FAST Conference Draws 20 Pharmaceutical Companies and Raises Over $2.2 Million for Research on Angelman Syndrome

DOWNERS GROVE, Ill., Dec. 8, 2020 /PRNewswire/ -- FAST (the Foundation for Angelman Syndrome Therapeutics) today announced that Virtually Unstoppable, their annual Summit & Gala benefitting Angelman syndrome (AS) raised over $2.2 million during their virtual two-day conference. The conference included two science conferences, an educational and best practices workshop, and a mini-Gala featuring award winning actor Colin Farrell. The event drew representatives from 20 global pharmaceutical companies. Researchers from 11 companies and academic institutions presented on their unique Angelman syndrome therapeutic approaches.

The science summit featured updates on multiple clinical trials, including two antisense oligonucleotide programs. The Translational Research Symposium and the Angelman Syndrome Outcome Measure and Biomarker Consortium (ABOM) featured presentations from the top scientists and industry leaders in this space on UBE3A gene isoform discoveries, real world data on the use of AS biomarkers and specific outcome assessment instruments, plus multiple potential gene therapy strategies, among other topics.

"The excitement at the FAST 2020 Conference was 'virtually' palpable! It's clear the passion and dedication of the community is what drives the truly innovative science and tremendous progress towards a treatment for individuals with Angelman syndrome. Ionis is proud to be part of such an amazing community," stated Rebecca Crean, Ph.D., Director, Clinical Development, Ionis Pharmaceuticals, Inc.

"I look forward to the FAST meeting every year, not only to connect with some of the best and brightest researchers in the field of AS, but also to hear the inspiring stories of AS families which remind me why I'm so lucky to be a part of this vibrant community," stated Rob Komorowski, Ph.D., Senior Clinical Scientist, Biogen.

"The FAST Virtual Summit is a collaborative initiative, engaging patients and families, as well as researchers and scientists, to work together toward finding a treatment for individuals with Angelman syndrome. It's truly incredible to see all that can be accomplished when passionate and collaborative minds come together. Taysha Gene Therapies is grateful to be a part of this dynamic community," stated Emily McGinnis, MPH, Chief Patient Officer and Head of Government Affairs, Taysha Gene Therapies.

"Seeing researchers, pharma and biotech representatives, and families from all over the world getting together at FAST was remarkable! The way I like to look at this is that we are all walking on a bridge of knowledge that needs to be built together step-by-step in order to bring a meaningful treatment to all families! It was a great and motivating experience. Thank you to FAST and all the families for their support!" stated Brenda Vincenzi, MD, Medical Director, PD Neuroscience, Roche.

The virtual platform allowed Angelman parents and caregivers an opportunity to interact, network and fundraise, despite Covid-19 challenges. "The astounding amount of funds raised during the event is a true testament to this community's dedication to improving the lives of individuals affected by Angelman syndrome," stated FAST Chairperson and GeneTx CEO Paula Evans.

"The weekend was 'virtually' incredible," stated FAST's CSO and GeneTx COO Allyson Berent, DVM, DACVIM. "Our global community listened to the most groundbreaking therapeutic advances in Angelman syndrome research, which has grown exponentially. All participants were impressed by the momentum and commitment of our academic, industry and clinical partners dedicated to finding meaningful treatments for those living with Angelman syndrome."

About Angelman Syndrome

Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. It is estimated to affect 1 in 12,000 to 1 in 20,000 people globally. Individuals with Angelman syndrome have developmental delays, balance issues, motor impairment, and debilitating seizures. Some are unable to walk and most do not speak. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome, suggesting that improvement of symptoms can potentially be achieved at any age.

About FAST

FAST (Foundation for Angelman Syndrome Therapeutics) is a Section 501(c)(3) nonprofit research organization singularly focused on funding research that holds the greatest promise of treating Angelman syndrome. FAST is the largest, nongovernmental funder of Angelman-specific research. Paula Evans, the mother of a girl with Angelman syndrome, founded FAST in 2008. In 2017, FAST formed GeneTx Biotherapeutics to develop GTX-102 for human clinical trials. More information can be found at www.CureAngelman.org.

Contact

Paula Evans
[email protected]
630-852-FAST

SOURCE FAST (the Foundation for Angelman Syndrome Therapeutics)