AVROBIO Announces Updated Clinical Data for AVR-RD-01 Gene Therapy in Fabry Disease

AVROBIO (Nasdaq:AVRO) a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose, today announced clinical data and patient updates from the investigator-sponsored Phase 1 study and the AVROBIO-sponsored Phase 2 clinical trial of AVR-RD-01. AVR‑RD‑01 is an ex vivo lentiviral gene therapy being investigated in Fabry disease.

As quoted in the press release:

The investigator-sponsored Phase 1 study is designed to assess the safety of AVR-RD-01 in up to six patients with Fabry disease who have been treated with standard of care enzyme replacement therapy (ERT) for at least six months prior to receiving AVR-RD-01. The Phase 1 study is conducted by the FACTs team (Fabry disease Clinical research and Therapeutics) in Canada and led by their principal investigator, Jeffrey A. Medin, Ph.D. The AVROBIO-sponsored Phase 2 trial of AVR-RD-01 (the FAB-201 Study¹) is an open-label, single-arm clinical trial evaluating the efficacy and safety of AVR-RD-01 in eight to twelve treatment-naïve patients.

“We are encouraged by the AGA enzyme activity we are seeing after treatment with AVR-RD-01 in the first two patients with Fabry disease in the Phase 1 study. Both of these patients have AGA activity that remains above the diagnostic range for males with classic Fabry disease, and all patients will continue to be followed for assessment of long-term durable response. We are especially pleased that patient 1 was taken off ERT in mid-July and remains off,” said Geoff MacKay, President and CEO of AVROBIO. “We recently achieved an important milestone in dosing the first patient in our ERT-naïve Phase 2 FAB-201 trial. This patient is now demonstrating AGA activity above the diagnostic range for males with classic Fabry disease three months after receiving our gene therapy treatment. In parallel, we continue our move towards implementation of a set of process optimization initiatives, including heightened vector efficiency, our fully closed, automated manufacturing system and conditioning.”

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