Our platform is based on our pioneering work with phosphorodiamidate morpholino oligomer—or PMO—chemistries, a versatile platform that may power the rapid design and development of new treatments for rare, infectious and other diseases.
PMOs: THE BASICS
PMOs are synthetic chemical structures modeled after the natural framework of RNA. While PMOs have the same nucleic acid bases found in RNA, they are bound to six-sided morpholine rings instead of five-sided ribose rings. In addition, the morpholine rings are connected to each other by phosphorodiamidate linkages instead of the phosphodiester linkages found in RNA.
Our core PMO chemistry is distinct from other approaches in RNA medicine: its unique physical properties possess favorable characteristics for drug development, including:
- SPECIFICITY PMOs have uncharged backbones and bind sequence-specifically to RNA targets
- STABILITY PMOs are highly resistant to degradation by enzymes
- VERSATILITY PMOs can be combined in a modular fashion to rapidly design and construct drug candidates that are specific for human or pathogen RNA and can be targeted to specific tissues
The PMO chemistry platform is highly adaptable, and we have developed next-generation PMO-based chemistries for advanced RNA-targeted therapeutics. These modified PMO-based chemistries are specifically designed to enhance tissue targeting, intracellular delivery, target selectivity and drug potency.
- PMOplus® Adds positionally specific positive molecular charges
- PPMOAttaches a peptide for
- PMO-X®Incorporates proprietary technology to fine-tune important physicochemical properties