Audentes Therapeutics program update for Pompe disease
Audentes Therapeutics presented pre-clinical work in support of an IND submission to study AT982 in both infantile and late onset Pompe disease patients.
AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease
Cambridge, MA, September 20, 2017 – AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following a Phase 1 Fabry program and a pre-Phase 1/2 Gaucher program.