Unique Drug Delivery System
- Novel, proprietary plasmid for use in gene therapy and biomolecule production
- Capable of carrying large genetic payloads
- High efficiency, lower cost production
- Under development for targeted neuromuscular diseases
Platform technology
- Modular gene therapy technology adaptable to numerous targets
- mCT gene therapy, based on temporary blockade of the Endoplasmic Reticulum Associated Degradation (ERAD) pathway
- Portfolio of patent protected, related but distinct compounds
- Supported by data in animals and cell lines
Initial focus on:
- Neuromuscular diseases: (TBA)
- CF and/or Lysosomal Storage Diseases:
- Orphan Drug Designation for Gaucher Disease
- Trans blood-brain-barrier applications accessing neuronal tissues,
- Intellectual Property protection: key patents issued and pending covering all technologies extending to 2044 and beyond.