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Astellas today announced interim safety data from the Phase 1/2 FORTIS trial of gene therapy AT845 in late-onset Pompe disease (LOPD)
FORTIS is an ongoing multicenter, open-label, dose-escalation, first-in-human clinical trial to determine the safety and tolerability of AT845 in adult patients with LOPD
As of the data cutoff date of December 3, 2021, 4 patients were enrolled in the trial, 2 patients received an infusion dose of 3 × 10E13 vg/kg (cohort 1), and 2 patients received an infusion dose of 6 × 10E13 vg/kg (Queue 2)
The data showed that AT845 was well tolerated in 4 adult patients with LOPD who were treated
Pompe disease (PD) is a rare, severe autosomal recessive metabolic disorder characterized by progressive muscle degeneration caused by mutations in the GAA gene that interfere with GAA protein production or function
AT845 is a muscle-directed gene replacement therapy using the AAV8 capsid serotype with cardiac and skeletal muscle specific promoters delivering a functional copy of the GAA gene
