- Phase 1-2
- Phase 3
H001 is an innovative non-viral gene therapy for increasing sialic production within the distal limb muscles, where GNEM patients need it the most. The advantage of H001 over viral vectors includes ability to re-dose while protecting vital internal organs from unnecessary exposure. Thereby, increasing expression of GNE within distal limb muscles and reducing the risk of adverse or potentially life-threatening side effects. The goal of this program is to stabilize arm/hand and leg/foot function. Critical scientific questions answered by the H001 program will pave the way for subsequent whole body gene therapy for GNEM.