Andrea Lobo, PhD,  science writer—

The first patient has been dosed in a Phase 3 trial evaluating the safety and efficacy of GTX-102, Ultragenyx Pharmaceutical’s experimental gene therapy for Angelman syndrome. The global Aspire study (NCT06617429) is expected to enroll about 120 children and adolescents, ages 4 to 17, with Angelman…

GTX-102, Ultragenyx’s investigational gene therapy for Angelman syndrome, improved cognition for participants in a Phase 1/2 trial after up to three years of treatment. Most patients also saw clinically meaningful improvements in one or more key areas, including communication, motor function, sleep, and behavior. The data support starting…

The Foundation for Angelman Syndrome Therapeutics (FAST) has teamed up with the University of Pennsylvania to develop an investigational gene therapy for Angelman syndrome. The nonprofit has sponsored preclinical research at the Gene Therapy Program at the University of Pennsylvania since 2017 to develop a gene therapy…

Changes in the levels of gut bacteria — the gut microbiome — are evident in small and larger animal models of Angelman syndrome, and these alterations may be related to patients’ gastrointestinal symptoms, a study reported. A notable difference between disease models and their healthy counterparts was seen in…