1st Angelman Patient in UK Dosed in Trial of Gene Therapy GTX-102

1st Angelman Patient in UK Dosed in Trial of Gene Therapy GTX-102

315500

1st Angelman Patient in UK Dosed in Trial of Gene Therapy GTX-102

A first patient in the U.K. has been dosed in the resumption of a Phase 1/2 clinical trial evaluating the safety of the gene therapy GTX-102 in children and adolescents with Angelman syndrome. The open-label study (NCT04259281), known as KIK-AS, is being run at Oxford University and is also underway in the U.S. and Canada. Its three sites are recruiting a total of up to 20 patients lacking the maternal UBE3A gene. Site and contact information can be found here. “We are…

You must be logged in to read/download the full post.