Startup AvroBio touts gene therapy candidates for blood cancer, Fabry disease
- Toronto-based startup AvroBio launched last week, announcing plans to begin phase 1 programs for two ex vivo gene therapies targeting acute myeloid leukemia and Fabry disease.
- AvroBio's programs are based on the work of scientists Christopher Paige and Jeffrey Medin. They are finishing up preclinical work on AVR-01 and AVR-02, and AvroBio hopes to move into phase 1 programs in early to mid-2016.
- AVR-01 is designed to trigger an immune response in patients with acute myeloid leukemia (AML). AVR-02 is intended to use a similar T-cell response to fight Fabry disease, a rare genetic lysosomal storage disease.
As noted by FierceBiotech, AvroBio's approach for AVR-01 is similar to the mechanism of action used in a therapy being developed by Ziopharma Oncology and in Bristol-Myer Squibb's skin cancer drug Yervoy.
The therapy works by genetically modifying the expression of the cytokine IL-12, an ultra-powerful immune signaling agent. Modified cells are re-infused, via a one-time procedure, and are supposed to then activate cytotoxic CD4-positive T cells to specifically target tumor cells.
AVR-02, on the other hand, is aimed at the rare Fabry disease, which affects roughly 11,000 people in the U.S. Currently the only treatment is Genzyme's enzyme replacement therapy, which requires bi-weekly infusions and costs $200,000 per year.
There is a dearth of treatment options for AML, which only has a 25% five-year survival rate. Assuming clinical trials move ahead successfully, this makes it more likely AvroBios could receive a designation from the FDA, such as a breakthrough therapy designation.