Audentes Therapeutics selects clinical development candidate for Pompe program
Audentes Therapeutics last night announced it has selected an optimized clinical development candidate for its Pompe disease program. Data supporting the selection were presented today during a general session at the 14th Annual WORLDSymposium 2018. "We are very excited to present the data highlighting the immunological and glycogen reduction benefits provided by our novel hybrid promoter vector in the Pompe disease mouse model. This innovative design reflects our commitment to gene therapy vector engineering and immunology, and strengthens our hope for the translation of AT982 into a transformative therapy for the Pompe patient community," stated John Gray, PhD, Senior VP and Chief Scientific Officer. Audentes plans to file an IND for AT982 in the second quarter of 2018 and plans to initiate a Phase 1/2 clinical study in the fourth quarter of 2018.