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…

Note: This story was updated Aug. 13, 2024, to correct the spelling of the measure of cognitive development to Bayley-4. Authorities in both the U.S. and Europe have provided positive feedback to Ultragenyx Pharmaceutical regarding plans for a Phase 3 clinical trial testing its gene therapy candidate GTX-102…

The Phase 1/2 KIK-AS clinical trial, which is testing the experimental genetic therapy GTX-102 in children with Angelman syndrome, has finished enrolling patients. With 74 enrolled, including 53 in dose-expansion groups, the therapy’s developer Ultragenyx Pharmaceutical said results will be available in the next few months. “With completion…

GTX-102, a gene therapy for Angelman syndrome, continues in a clinical trial to be safe and show benefit when taken for over a year, with the longest period being nearly two years, according to its developer, Ultragenyx Pharmaceutical. No new serious side effects or reports of leg weakness…

Ultragenyx Pharmaceutical has been cleared by the U.S. Food and Drug Administration to proceed to higher dosing of its investigational gene therapy GTX-102 in children and adolescents with Angelman syndrome in a Phase 1/2 clinical trial. The amendment to the KIK-AS trial (NCT04259281) protocol in the…

Because antisense oligonucleotide (ASO) therapies target specific genetic sequences, differences across mammals make it challenging to translate potential Angelman ASO-based therapies from preclinical models to the clinic. GTX-102, an investigational ASO therapy for Angelman syndrome, was specifically designed to be effective in patients and relevant animal models, however.

Ultragenyx will continue clinical development of GTX-102, an investigational treatment for Angelman syndrome, after acquiring GeneTx Biotherapeutics — the company that was developing it — in a $75 million deal earlier this month. The therapy was originally developed in the laboratory of Scott Dindot, PhD, in the…

GeneTx Biotherapeutics, a company set up by the Foundation for Angelman Syndrome Therapeutics (FAST), has been acquired by Ultragenyx Pharmaceutical, which will take over the ongoing GTX-102 clinical program for Angelman syndrome. The two companies began working together in 2019, and they jointly launched a Phase…

The investigational gene therapy GTX-102 shows potential to lessen disease severity and improve function in children and adolescents with Angelman syndrome, according to interim data from a Phase 1/2 clinical trial. Six-month follow-up data for 11 patients showed that treatment with GTX-102 resulted in improvements across exploratory…