First international council to support accelerated drug development for Angelman syndrome and related neurodevelopmental disorders
DOWNERS GROVE, Ill., Feb. 19, 2021 (GLOBE NEWSWIRE) — FAST (Foundation for Angelman Syndrome Therapeutics) and the Simons Foundation Autism Research Initiative (SFARI) today announced a collaboration to launch the International Angelman Syndrome Research Council (INSYNC-AS). Using a robust integrated approach, the Council will evaluate and drive research initiatives in Angelman syndrome (AS) and other neurodevelopmental disorders (NDDs).
INSYNC-AS’s goal is to build community and leverage the combined skills of world leaders in neuroscience, clinical research, genetics, drug development, regulatory expertise, and other thought leaders, in order to accelerate drug development for Angelman syndrome and other related disorders. The Council was formed to be an innovative model that encourages research in areas where gaps need to be filled. A multi-functional combination of skill sets and expertise will help to advance FAST’s deep funding strategies, while leveraging these learnings to other NDDs.
“The emergence of novel therapeutic platforms such as gene therapy and genome editing has created exciting opportunities for possible treatments for neurodevelopmental disorders. INSYNC-AS will be a great way to help the Angelman syndrome community assess, and potentially direct, these technologies to therapies,” said James Wilson, MD, PhD, a professor and director of the Penn Gene Therapy Program and Penn Orphan Disease Center, a member of the Council.
This Council will take the cutting-edge scientific discoveries available in Angelman syndrome research to the next level by promoting and growing the work being pursued by our current thought leaders, while advancing strategies that are not yet being implemented. By sharing knowledge and clinical expertise, the Angelman syndrome community can be sure that for AS, and other similar NDDs, world leaders are working to the highest quality through state-of-the-art initiatives.
Dr. John Spiro, interim director of SFARI, stated, “SFARI is excited about partnering with Dr. Allyson Berent, and the entire Angelman syndrome community, to capitalize on what they have learned from their successes in bringing potential therapeutics to individuals with AS. SFARI hopes to keep the momentum going to make those treatments even more effective and durable, and to extend the lessons learned to other neurodevelopmental disorders with known genetic causes. FAST’s sense of urgency and laser focus on moving findings from the lab into clinical trials is an inspiration to all of us who work in this field.”
“The inspiration to establish the INSYNC-AS Consortium was to further all translational research avenues for Angelman syndrome, leaving no stone unturned, highlighting FAST’s mission in every way,” stated Dr. Allyson Berent, FAST’s Chief Science Officer. “This is beyond exciting for all of those living with Angelman syndrome, and hundreds of other neurodevelopmental disorders. This collaboration between FAST and SFARI is just spectacular for our entire AS community and hundreds of other neurodevelopmental disorders.”
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, mother of a girl with Angelman syndrome, founded FAST in 2008. In 2017, FAST formed GeneTx Biotherapeutics to develop GTX-102, and antisense oligonucleotide, for human clinical trials. More information can be found at www.CureAngelman.org.
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 currently no 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.
SFARI’s mission is to improve the understanding, diagnosis and treatment of autism spectrum disorders by funding innovative research of the highest quality and relevance. Since its launch in 2006, SFARI has supported over 550 investigators doing autism-related research in the U.S. and abroad. Research projects include studies at the genetic, molecular, cellular, circuit and behavioral levels, in addition to clinical and translational studies.
Allyson Berent, DVM, DACVIM