Avrobio shares positive data on rare disease gene therapy

Dec. 7, 2022

Avrobio announced new compelling clinical data from five patients with Gaucher disease after they received a single dose of the Massachusetts-based biotech's investigational hematopoietic stem cell (HSC) gene therapy. 

The interim pharmacokinetic, pharmacodynamic and clinical efficacy data showed stabilization or reversal of multiple clinically relevant measures in the first-ever Gaucher disease type 3 (GD3) patient and four Gaucher disease type 1 (GD1) patients dosed with AVR-RD-02.

Gaucher disease is the largest, most common lysosomal disorder. The current standard of care is enzyme replacement therapy but even with this, people with GD1 typically have a shortened life expectancy and may experience debilitating symptoms.

The trial data is good news for Avrobio, after halting its Fabry disease program earlier this year after disappointing trial data. At the time, the company said it was shifting focus to other clinical-stage programs in its lysosomal disorder pipeline — and that decision appears to have paid off.

Now, following constructive meetings with the FDA and MHRA, Avrobio says it plans to initiate a phase 2/3 pediatric clinical trial for AVR-RD-02 in GD3 in the second half of 2023.