The Foundation for Angelman Syndrome Therapeutics, known as FAST, is partnering with the Simons Foundation Autism Research Initiative (SFARI) to launch the International Angelman Syndrome Research Council — or INSYNC-AS — with a goal to “build community and leverage the combined skills of world leaders in neuroscience, clinical research, genetics, drug development, regulatory expertise, and other thought leaders.”
“This collaboration between FAST and SFARI is just spectacular for our entire AS community and hundreds of other neurodevelopmental disorders,” said Allyson Berent, the foundation’s chief science officer.
The council was formed, according to the two groups, to be an innovative model for encouraging research “in areas where gaps need to be filled.”
“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,” John Spiro, PhD, interim director of SFARI, said in another press release.
In 2017, FAST launched GeneTx Biotherapeutics, a biotech company devoted to the development of an antisense oligonucleotide (ASO) called GTX-102. The GeneTx Bio investigational compound is designed to target the molecule — known as UBE3A antisense transcript, or UBE3A-AS — that causes Angelman and to prevent it from working.
GTX-102’s safety and tolerability are being evaluated in a clinical trial, called KIK-AS, or Knockdown of UBE3A-antisense in Kids with Angelman Syndrome (NCT04259281).
SFARI is a scientific initiative within the Simons Foundation that supports scientific projects in autism spectrum disorders.
The partnership between these two foundations allowed the launch of INSYNC-AS, a research consortium that will use an integrated and collaborative approach to leverage translational research in the development of new therapies for Angelman and 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,” said Jim Wilson, MD, PhD, professor and director of the Penn Gene Therapy Program and Penn Orphan Disease Center.
“INSYNC-AS will be a great way to help the Angelman syndrome community assess, and potentially direct, these technologies to therapies,” said Wilson, a member of the council.
One of the council’s goals is to reach a consensus on the best way to test new candidate therapies in pre-clinical studies.
“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,” Spiro added.
Through an annual conference, INSYNC wants to bring together speakers and world leaders to promote Angelman research.
“The inspiration to establish the INSYNC-AS Consortium is to further all translational research avenues for Angelman syndrome, leaving no stone unturned, and highlights FAST’s mission in every way,” Berent said.