Durable. Repeatable. Outpatient.
A cell-based gene therapy for Fabry, Gaucher, Pompe and other enzyme deficiency disorders
Clinically demonstrated.
Five Fabry patients treated.
5 years freedom from biweekly infusions.
Pilot clinical trial complete in Canada with U.S. Phase 2 in preparation.
IND filing targeted Q1 2027 with trial start expected in late 2027.