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FAST Highlights Research Advancements at 2021 Global Science Summit and Gala

FAST, the Foundation for Angelman Syndrome Therapeutics, hosts annual hybrid event focused on science and pharmaceutical advancements for Angelman syndrome

DOWNERS GROVE, Ill. (Dec. 15, 2021) — FAST, the Foundation for Angelman Syndrome Therapeutics, announces the success of their recent hybrid Global Science Summit and Gala. The annual Global Science Summit and Gala, which took place on December 3rd – 4th 2021 in Austin, Texas, featured over 20 presentations from researchers, clinicians and pharmaceutical companies.

The event featured updates on Angelman syndrome research programs and clinical trials from the top scientists and industry leaders, educational workshops for caregivers, therapists, and teachers, and helpful vendors that provided Angelman families with the necessary goods and services to support their loved ones living with Angelman syndrome.

Dr. Jan A. Nolta and Dr. David Segal, both from the University of California, Davis School of Medicine, were keynote speakers at the 2021 Global Science Summit. The event also featured many notable research and pharmaceutical updates, including:

  • Dr. Jim Wilson from Perelman School of Medicine at the University of Pennsylvania announced he is working on four strategies for gene therapy in Angelman syndrome, including a study on hUBE3A that has shown positive results on early toxicology studies.
  • Drs. Joe Anderson and Mehrdad Abedi from University of California, Davis School of Medicine shared exciting gene therapy technology involving autologous hematopoietic progenitor cell gene therapy, or HPC-GT. They announced this is currently in IND-enabling studies with a goal to commence a clinical trial for human application in both adults and pediatrics with Angelman syndrome in the near term.
  • Dr. Allyson Berent, chief science officer of FAST, unveiled FAST’s Accelerated Roadmap for a Cure, including the Angelman Syndrome Search and Rescue Mission, a new initiative to identify everyone in the world living with Angelman syndrome. This will ensure that the global community is strengthened by FAST’s vision, where knowledge is power and patient access to all future potential therapies is a priority.
  • Dr. Becky Crean from Ionis Pharmaceuticals announced Ionis HALO, a new clinical trial. Ionis HALOS has been cleared by the FDA to begin a Phase 1 clinical trial for an IT delivered ASO for a potential treatment for Angelman syndrome.
  • Dr. Scott Stromatt from GeneTx Biotherapeutics, in collaboration with the programs primary investigator, Dr. Elizebeth Berry-Kravis from Rush University, noted that GTX-102 showed evidence of clinical improvement in multiple domains in the first cohorts treated with promising updates on Vineland Adaptive Behavior Scale-3 in those patients. The newly shared clinical improvements included communication, socialization, personal living skills, and gross and fine motor skills.
  • Dr. Brenda Vincenzi from Roche Pharmaceuticals provided a recruitment update for TANGELO. There are 43 participants enrolled with no dropouts. The 1-year-old to 4-year-old cohorts should start recruitment in the United States in the first half of 2022, while the older cohorts (5 to 12 years old) are fully enrolled globally.
  • Dr. Nancy Jones from Neuren Pharmaceuticals shared an update on NNZ-2591, an analog of cyclic glycine-proline (cGP), a metabolite of IGF-1. The drug has shown to improve behavior, cognitive, motor deficits and seizures in the AS mouse model. The Phase 2 trial for AS will soon launch in Australia.

The annual event was an opportunity for the Angelman syndrome community to connect face-to-face, both in person and virtually, to learn about the astonishing science and pharmaceutical advancements, and raise a significant amount of money to advance many programs for Angelman syndrome forward in parallel. The two-day event concluded with a gala featuring a keynote by Dr. Edwin Weeber, a live performance from Casey McPherson of Alpha-Rev, and rare disease advocate, and the show-stopping entertainment from Skyrocket.

The 2021 Global Science Summit and Gala was made possible through the generous support of sponsors, including top-level sponsors, Keder Solutions, Ultragenyx, Quincy’s Quest Foundation, PTC Therapeutics, Thomas Financial, the Goelz family and Biogen.

For more information on the Global Science Summit and Gala, please visit http://fast-gala.org. For more information on the Foundation for Angelman Syndrome Therapeutics (FAST), please visit www.cureangelman.org. Keep up with the latest news from FAST on Facebook, Twitter, Instagram and LinkedIn.