AVR-RD-03 / AVROBIO - LARVOL DELTA

AVROBIO Reports Second Quarter 2020 Financial Results and Provides Business Update (Businesswire) - "AVROBIO’s research program for Pompe disease, AVR-RD-03, is currently advancing a candidate through pre-clinical studies. Data presented at ASGCT in May 2020 showed the ability of AVROBIO’s optimized lentiviral vectors, in combination with a proprietary Glycosylation-Independent Lysosomal Targeting (GILT)-tag technology, to demonstrate significant glycogen reduction in both the muscle and central nervous system of a Pompe disease mouse model. A preclinical Investigational New Drug (IND)-enabling proof-of-concept study is currently underway and expected to conclude in 2020."

Preclinical • Genetic Disorders • Pompe Disease