Ultragenyx Pharmaceutical Ultragenyx announces completion of enrollment in Global Phase 1/2 Trial of GTX-102
Ultragenyx announced the completion of enrollment in Global Phase 1/2 Trial of GTX-102 in pediatric patients living with Angelman syndrome and data from at least 20 patients enrolled in dose expansion cohorts anticipated in the first half of 2024. Read press release here.
FAST to Advance Gene Therapy Candidate through IND-Enabling Studies Conducted in Partnership with the University of Pennsylvania
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 rare neurogenetic disorder that is estimated to impact approximately […]
The National Institutes of Health awarded the first phase of a roughly $40M grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases
In October 2023, the National Institutes of Health awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome. The project team named under […]
An update on Roche’s trials of rugonersen (RO7248824) in Angelman syndrome
We are sad to report that Roche has decided they will not initiate any new trials of rugonersen (RO7248824) in Angelman syndrome. If you are currently participating in the study, you should be hearing from the Investigator or their team at the site where your child is enrolled regarding important information about next steps for your […]