- 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 distal muscle function. Critical scientific questions answered by H001 program will pave the way for subsequent AAV based gene therapy for GNEM.