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.

pmo_vs_rna

 

PMO CHARACTERISTICS

Our core PMO chemistry has unique characteristics that are favorable 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

NEXT-GENERATION CHEMISTRIES

 

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

PPMO

Attaches a peptide for enhanced delivery

PMO-X®

Incorporates proprietary technology to fine-tune important physicochemical properties

Therapeutic Applications
Manufacturing
Strategic Partnerships