INSYNC-AS: A collaboration between FAST and the Simons Foundation Autism Research Initiative (SFARI)

INSYNC-AS: A collaboration between FAST and the Simons Foundation Autism Research Initiative (SFARI)

The Foundation for Angelman Syndrome Therapeutics (FAST) is here to demonstrate our commitment to identifying and furthering scientific advancements for various potential therapeutics that treat Angelman syndrome (AS) through this exciting collaboration with the Simons Foundation Autism Research Initiative (SFARI). The launch of the International Angelman SYNdrome Research Council (INSYNC-AS) will actively evaluate and drive research initiatives in Angelman syndrome and other similar neurodevelopmental disorders (NDDs) using a robust integrated approach by tapping into the expertise and experience from the foremost thought leaders in diverse fields. This Council will not only include experts focusing on Angelman syndrome currently, but will bring in thought leaders working in many other arenas in order to further grow our translational research roadmap.


The goal of INSYNC-AS is to build a community of collaborative advisors by leveraging the combined skill set of the scientists, clinicians, geneticists, pharmaceutical executives, and other thought leaders, in order to empower further drug development for Angelman syndrome. This Council of Excellence, or “Brain Trust”, will provide innovative ideas, ensure potential research avenues are identified, explore novel therapeutic platforms and further de-risk those heavily invested, while encouraging new research areas where gaps need to be filled, and consider a multi-functional combination of skill sets and expertise to help prioritize FAST’s deep funding strategies, while leveraging these learnings to other NDDs.

Another goal of this consortium is to encourage strong and clear consensus amongst pre-clinical colleagues working in neurodevelopmental disorders (NDD) of the best way to clinically test novel therapeutic compounds to ensure neurobehavioral testing is consistent, of the highest quality, and integrating the latest strategy in regulatory practice.


By sharing knowledge and clinical expertise among the best researchers in the world, industry leaders and regulatory experts, FAST can be sure that Angelman syndrome, and other similar NDDs, are working to the highest quality through state-of-the-art initiatives. INSYNC will not only impact treatments for AS, but will also impact the drug development process for other NDDs and patient group-led research portfolios.  INSYNC-AS was created to be an innovative model on how FAST considers all research strategies, with the ultimate goal of funding translational measures to benefit the lives of those living with Angelman syndrome.


Since 2008, when FAST launched, the investment into research and the development of meaningful therapeutics has increased exponentially.  In the past 10 years FAST has robustly invested over $20MM in numerous discovery programs and 9 gene-altering, gene-replacement, and/or disease modifying platforms. In addition, in 2017 FAST launched a new biotech, GeneTx Biotherapeutics, to pursue an antisense oligonucleotide for the potential treatment of AS. This program allowed the first ASO clinical trial in AS to begin in February of 2020. While this journey has been exciting, we realize that our work is just beginning, and we are here to support all therapeutic platforms that may bring meaningful change to the lives of all individuals living with Angelman syndrome, and to make sure that these learnings can be translated to those living with hundreds of other NDDs.

In a recent press release FAST’s Chief Science Officer, Dr. Allyson Berent states, “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. 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.”

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.

Dr. John Spiro, interim director of SFARI, states, “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.”

INSYNC will work collaboratively through a focused yearly conference to dive into the scientific and translational research needs in Angelman syndrome. Invited speakers and world leaders will work together to ensure the Angelman syndrome research is ahead of publications, and not behind.

As stated by Dr. Jim Wilson, MD, PhD, a professor and director of the Penn Gene Therapy Program and Penn Orphan Disease Center, a member of the Council. “The emergence of novel therapeutic platforms such as gene therapy and genome editing has created exciting opportunities for possible treatments for neurodevelopmental disorders. The INSYNC-AS will be a great way to help the Angelman syndrome community assess, and potentially direct, these technologies to therapies.”

We are so thrilled to have some of the greatest leaders in the field support such an initiative.