FAST joins with University of Pennsylvania to develop investigational AAV gene therapy for Angelman syndrome

Reviewed by Lily Ramsey, LLMOct 17 2023

The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS).

Angelman syndrome is a nondegenerative neurogenetic disorder that is estimated to impact approximately 1 in 15,000 births, or potentially 500,000 individuals world-wide, and is associated with a significant clinical unmet need including: developmental delay, lack of verbal speech, seizures, profound sleep disorder, and motor impairments, among other serious symptoms. There is currently no approved therapy to treat Angelman syndrome in any country.

FAST has sponsored preclinical research studies at the Gene Therapy Program (GTP) at the University of Pennsylvania (Penn) since 2017 towards the development of a central nervous system (CNS)-delivered gene therapy for Angelman syndrome. A robust package of preclinical data now support the further advancement of a human clinical candidate through the final investigational new drug application (IND)-enabling studies that are required for subsequent first-in-human clinical trials. This updated and expanded collaborative agreement builds upon the original research funding provided by FAST to Penn and enables FAST to work more closely in partnership with the Penn team to further accelerate the advancement of the candidate towards a clinical trial for individuals living with AS.

We have been working toward this 'go or no-go decision' since 2017. To see the promising results of this robust preclinical data package, based on research that FAST has been so diligently funding for years, is incredibly gratifying. Too many companies over the past year have been shifting their priorities in the rare disease space, but the priority for FAST will never change. It is our job to ensure that excellent science is always advanced as safely and efficiently as possible, and that we seize every opportunity to potentially benefit those living with Angelman syndrome."

Allyson Berent, DVM, DACVIM, Chief Science Officer of FAST

"Our collaboration with the team at FAST, and the entire Angelman syndrome community, has been incredibly productive and rewarding, since its inception," said Jim Wilson, MD, PhD, director of the Gene Therapy Program, the Rose H. Weiss Professor and director at the Orphan Disease Center, and a professor of Medicine and Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. "Our two teams have been both mission and data driven, working closely together to reach this critical inflection point, and FAST and GTP have made an instrumental decision on a promising human clinical candidate to further advance this gene therapy forward quickly and safely for the potential treatment of Angelman syndrome."