First Patient Dosed in FORTIS Trial of AT845 for LOPD

First Patient Dosed in FORTIS Trial of AT845 for LOPD

291708

First Patient Dosed in FORTIS Trial of AT845 for LOPD

The first patient has been dosed in the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD). AT845 is designed to deliver a healthy copy of the gene that encodes the enzyme acid alpha-glucosidase (GAA) directly to muscle cells. People with Pompe lack functional GAA, leading to the toxic accumulation of the sugar molecule glycogen in cells, most notably muscle cells. FORTIS (NCT04174105), which is expected to enroll…

You must be logged in to read/download the full post.