Our experienced R&D team has developed and utilized our proprietary technology platform to produce several promising siRNA therapies targeting different genes for different indications and target organs. Using our accumulated know-how and expertise, we are developing therapies for unserved or underserved diseases involving the kidneys, eyes, central nervous system, ears, lungs, and more.
Our siRNA platform is comprised of proprietary siRNA selection software, proprietary siRNA structural features, and a proprietary toolbox of chemical modifications. Combined with the team’s expertise and know-how, this platform allows to generate unique siRNA compounds with improved pharmacological properties.
Our siRNA drug candidates:
- Are highly active in eliciting inhibition of target gene expression
- Have a favorable safety profile
- Highly specific to the target
- Avoiding hybridization dependent and independent (e.g., immunostimulatory) off-target effects
- Are self-delivering (can enter the cells and inhibit gene expression without the need for complex formulation)
- Have adaptable duration of effect – from days to months, depending on therapeutic needs
- Are free from third-party intellectual property coverage.