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3rd annual INSYNC-AS Meeting recap

This past week the FAST team, SFARI (Simons Foundation Autism Research Initiative), industry sponsors, regulatory experts and 22 of the INSYNC-AS Council members, along with invited speakers and representatives from other rare neurodevelopmental disorder groups, met in person in New York City for the 3rd Annual International Angelman Syndrome Research Council (INSYNC-AS) meeting.

INSYNC-AS is a consortium co-sponsored by FAST and SFARI, bringing together a team of diverse global experts designed to actively evaluate and help drive research initiatives in Angelman syndrome and other NDDs (neurodevelopmental disorders) by tapping into the expertise and experience of the foremost thought leaders in various fields to further grow FAST’s translational research roadmap. The goal is to build a Brain Trust (a community of collaborative advisors) in order to do a gap analysis on where we are, explore novel therapeutic platforms that we have not yet supported, tackle therapeutic challenges, and de-risk and bolster initiatives that are already ongoing.

The focus of INSYNC-AS this year was on innovative clinical trial design for rare neurodevelopmental disorders, with a focus on the clinical trial landscape for Angelman syndrome. Key topics included considerations on the potential to share control data, and leveraging robust natural history data in trial design and conduct. One area we focused on was how to best utilize innovative statistical analyses and novel clinical trial designs, which could help to limit the need for large numbers of patients in trials, especially in the control arms of a study. Experts in the field provided examples of innovative trial designs that have been used in studying other rare disease populations.  One idea included developing a master protocol where multiple therapies can be evaluated simultaneously versus a common control group to ensure more individuals with AS are on active drug rather than a placebo in these trials. Master protocols have been used in many clinical trials ranging from oncology to ALS and are gaining momentum given their advantages in having an existing platform to more quickly evaluate the safety and efficacy of a drug without having to design a new protocol for every study. With the robust pipeline in Angelman syndrome, it is a good time to think about collaborative efforts to streamline development. Experts working in dozens of other rare NDDs were present, helping to deepen the conversation through their own learned experiences.  We also discussed other serious challenges facing the field like the need for improved delivery methods for gene therapy in neurologic disorders and how we can work together to understand what early-stage animal model or cell line data is necessary to most efficiently make to best go- and no-go decisions.

Also in attendance at this year’s INSYNC meeting were representatives from other rare neurodevelopmental disorder groups including Rett syndrome, CDKL5 Deficiency Disorder, and STXBP1 Disorder. After hearing updates from each of these communities and scientific leaders on their own progress in drug development, it was abundantly clear how many commonalities all of these groups share and how much faster and more efficiently we can move our own individual pipeline programs forward if we work together. Meetings like INSYNC allow us to collaborate across rare disorder communities and help each other navigate the clinical trial landscape, sharing tools and approaches that have been successful for others and thinking more broadly to make a bigger impact on Angelman syndrome. It was an incredibly, insightful and invigorating meeting that will help accelerate not just the Angelman syndrome therapeutic landscape, but other rare neurodevelopmental disorder communities, all with a similar goal: to bring meaningful change to the lives of all individuals living with these NDDs.