Avrobio Shares Rally On Fabry Disease Gene Therapy Encouraging Benefit In Early-Stage Study

Avrobio Inc (NASDAQ: AVRO) has reported positive data from the ongoing Phase 2 FAB-GTi Phase 1/2 trial evaluating AVR-RD-01, an ex vivo lentiviral gene therapy for Fabry disease. 100% reduction, or complete clearance, of the toxic substrate in the kidney biopsy, was observed in the first patient dosed.
The company also announced six-month data from the first patient dosed in the Phase 1/2 trial of AVR-RD-02, an ex vivo lentiviral gene therapy for Gaucher disease type 1, showing substantial improvement over pre-gene therapy enzyme replacement therapy baseline in key biomarkers at six months post-gene therapy. Plasma chitotriosidase levels decreased 49%, and toxic metabolite lyso-Gb1 levels dropped 44% in the first patient at six months post-gene therapy, compared to the patient's pre-gene therapy ERT baseline levels.
Additionally, preliminary data from the investigator-sponsored Phase 1/2 trial of AVR-RD-04 showed that all three cystinosis patients demonstrated strong data across multiple measures, including substantial improvement in photophobia in the first patient 12 months post-gene therapy. All patients remained off cysteamine pills, and eye drops, with trial enrollment completion expected this year.
The Investor and analyst presentation is scheduled today at 8 a.m. ET.
Price Action: AVRO jumped 19.6% at $20.46 in premarket trading on the last check Monday.