SPY363.22+5.76 1.61%
DIA300.43+4.54 1.53%
IXIC12,036.79+156.15 1.31%

AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipeline

Three months post-gene therapy, first patient in Gaucher trial shows reductions in the toxic metabolite plasma lyso-Gb1 and plasma chitotriosidase as compared to baseline when the patient was on ERT Ongoing Fabry

· 11/17/2020 07:02

Three months post-gene therapy, first patient in Gaucher trial shows reductions in the toxic metabolite plasma lyso-Gb1 and plasma chitotriosidase as compared to baseline when the patient was on ERT

Ongoing Fabry disease trials continue to demonstrate sustained durability, with first patient out 3.5 years

One year post-gene therapy, first patient in cystinosis trial remains off cysteamine, with positive data across multiple measures, including substantial reduction in cystine crystals in cornea

Clinical trial recruitment gaining momentum with five new patients expected to be dosed, enrolled or consented in 4Q 2020

Gaucher disease type 3 program added to pipeline; recently added Hunter syndrome program planned to enter clinic next year

Virtual R&D Day to be webcast today starting at 9 a.m. ET