CAMBRIDGE, Mass.–(BUSINESS WIRE)–Feb. 6, 2019– AVROBIO, Inc. (Nasdaq: AVRO) (the “Company”), a Phase 2 clinical-stage gene therapy company, today announced clinical trial updates from the ongoing investigator-sponsored Phase 1 clinical study and the AVROBIO-sponsored Phase 2 clinical trial of AVR-RD-01. AVR-RD-01 is an investigational gene therapy candidate designed to treat Fabry disease by inserting the GLA gene that encodes functional α-galactosidase A (AGA, the enzyme that is deficient in patients with Fabry disease) with the goal of enabling continuous endogenous AGA production and distribution to tissues and organs.
“Today’s update shows a consistent readout across multiple surrogate markers that support the potential of AVR-RD-01 to address the genetic basis of Fabry disease,” said Birgitte Volck, MD, PhD, AVROBIO’s President of Research and Development. We believe these results are significant because the data reported show sustained AGA enzyme levels with associated reductions in substrate and metabolite levels. This suggests that our gene therapy exerts its effects as intended in patients previously treated with ERT, as well as in treatment-naïve patients. Taken together, these clinical data represent a growing body of evidence of the therapeutic potential of AVR-RD-01 as a gene therapy for patients with Fabry disease.”