Therapeutic Pipeline

  • Discovery
  • Pre-Clinical
  • Phase 1-2
  • Phase 3
  • Market

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 path towards subsequent whole body gene therapy for GNEM.


A next generation non-viral vector that benefits from the advantages of H001 combined with improved therapeutic gene expression.


H002 is an AAV vector designed for better targeting of muscles of GNEM and more effective Sialic production. Therapeutic gene expression is driven by tissue-specific promoter.