GeneTx, Ultragenyx announce IND application active for GTX-102
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical (RARE) announced that GeneTx's Investigational New Drug, or IND, application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome, filed with the FDA is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020. "The FDA's clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community," said Paula Evans, CEO at GeneTx. "Assessing an investigational therapy that aims to address the underlying cause of this devastating disorder has the potential to meaningfully alter the clinical course of patients' lives." "Based on preclinical data, we believe that GTX-102 offers tremendous promise and may one day provide patients with a potentially transformative therapeutic option," said Camille Bedrosian, Chief Medical Officer of Ultragenyx. "We look forward to continuing our partnership with GeneTx to address this devastating disorder for which there are currently no approved treatments."