Audentes Therapeutics presented pre-clinical mouse-model data at the American Society of Gene & Cell Therapy Annual Meeting in May, 2018. Their pre-clinical work is in support of an investigational new drug (IND) submission to study AT982 in both infantile and late onset Pompe disease patients. They anticipate submitting the IND in 2019. Audentes also plans to start a natural history run-in study for both infantile and late onset Pompe disease, also planned for 2019.
Read more about Audentes Therapeutics’s gene therapy program for Pompe disease.
Tagged: gene therapy, preclinical