Shots:

• The P-I/II (STAAR) study evaluates ST-920 in patients with Fabry disease. As of cutoff date, elevated α-Gal A activity was maintained for 4 patients treated in 2 dose cohorts (0.5e13/1e13vg/kg)
• Patients in 1st dose cohort have maintained elevated activity for 1yr. & in long-term follow-up study now. In 3rd dose cohort, 5th patient exhibited activity within mean normal α-Gal A levels @2wk.
• The therapy was well tolerated in 3 dose cohorts in 5 treated patients, no patients experienced liver enzyme elevations requiring steroid treatment, improvements in the ability to sweat in 3 patients, no progression of Fabry cardiomyopathy, ~40% reduction in biomarker in patients with elevation in plasma lyso-Gb3

Ref: Businesswire | Image: Dome Construction