
Gene Therapy for Fabry Disease to Advance to Phase 2 Clinical Trials
Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).
Biotechnology company Avrobio has
FD is caused by absent or deficient activity of the lysosomal enzyme alpha-galactosidase A (a-Gal A) (encoded by the GLA gene), and causes buildup of globotriaosylceramide in cells, which can result in irreversible organ damage. Avrobio’s proposed treatment, AVR-RD-01, is created by extracting and isolating CD34+ stem cells from patients with FD, then transducing them with lentiviral vector that carries a normal GLA gene. The resulting product is infused back into the patient in an out-patient setting in a single dose.
The company will use the newly raised funds to initiate a phase 2 trial of AVR-RD-01 based on promising initial
If eventually approved, AVR-RD-01 could revolutionize the treatment of FD; currently, the standard of care for the disease is enzyme replacement
In addition to AVR-RD-01, Avrobio hopes to develop 3 more gene therapies targeting other lysosomal storage disorders: Gaucher disease, and cystinosis (for both of which Avrobio hopes to initiate clinical development by mid-2019), as well as Pompe disease.
Newsletter
Stay ahead of policy, cost, and value—subscribe to AJMC for expert insights at the intersection of clinical care and health economics.