FAST Unites Global Research Experts for First INSYNC-AS Meet

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by Mary Chapman |

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INSYNC-AS/angelmansyndromenews.com/researchers hold first global meeting

The inaugural meeting of the International Angelman Syndrome Research Council, known as INSYNC-AS, focused on the translational research landscape in Angelman syndrome (AS), and on prospects for new treatments.

The recent assembly of Angelman scientists and experts from around the world was pulled together by the Foundation for Angelman Syndrome Therapeutics, or FAST, in partnership with the Simons Foundation Autism Research Initiative, known as SFARI.

INSYNC-AS is comprised of global experts in genome sequencing research, endpoint development, clinical trials, and regulatory affairs. The event brought together representatives of various aspects of the translational research continuum to discuss FAST’s mission, which is to discover therapies for all Angelman patients.

Translational study takes discoveries made in the lab, clinic, or in the field and transforms them into new therapies and approaches to care.

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“INSYNC-AS created an opportunity for us to unite global experts to share the current landscape of translational research in Angelman syndrome,” Allyson Berent, FAST’s chief science officer, said in a press release. “Each of the council members in attendance were tremendously participatory and focused on the vision of the initiative to further translational science.”

Among the presentations at the day-long event was a key talk on neurogenetics — a field of scientific research that uses genome sequencing advances to better understand the causes of brain and nerve disorders. Attendees also learned, in other sessions, about new therapeutic programs, treatment delivery options, and clinical endpoints and biomarker development for Angelman syndrome.

Each presentation was followed by a discussion aimed at advancing translational research initiatives.

“The team was impressed with what has been accomplished in such a short period through FAST’s research initiatives and robust funding strategies,” Berent said. “The mission of FAST, which is to bring meaningful therapeutics to all individuals living with Angelman syndrome, was the guiding light for the entire conference.”

By the end of the meeting, Berent said, “it was clear … that our mission at FAST was not only possible, but it is probable.”

“The future has never looked more positive for the development of transformative treatments for people with Angelman syndrome,” said Omar Khwaja, MD, chief medical officer at Vectivbio, a biotechnology company in Switzerland.

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“The INSYNC-AS initiative brings together a truly cross-disciplinary group of experts from across academia, industry, and the Angelman community to ensure we are rapidly identifying opportunities, solving gaps, prioritizing valuable resources, and accelerating the development of the most promising therapeutic approaches,” Khwaja said.

“I was very honored to be part of such a creative and committed group who are undaunted by the challenges ahead and optimistic for the day when Angelman syndrome is treatable,” he added.

Current treatments focus on controlling the seizures commonly associated with Angelman, and managing the disease’s physical and behavioral symptoms.

“The INSYNC-AS event brought together experts in AS from preclinical, clinical, and industry perspectives and sat them all down together to talk about one thing: how do we get treatments to people who need them as quickly and as safely as possible,” said David Segal, PhD, a professor at the University of California Davis MIND Institute. “I think we all left with a better understanding of how to work with each other to make this happen.”

FAST and SFARI, a research program that focuses on autism research, plan to enhance INSYNC-AS to support other neurodevelopmental disorders by outlining roadmaps and necessary steps within the realm of translational research.

Berent said that Angelman syndrome “will serve as a model for so many other rare disorders.”