The Science Behind SeleRNA
Cardiomyocyte-selective modRNA translation to transform cardiac care
Cardiomyocyte-Selective modRNA Translation System: CM-SMRTs
Our platform harnesses cardiomyocyte-selective translation to enable therapeutic protein expression directly within cardiac muscle cells. This approach. turns cardiomyocytes into localized producers of therapeutic protein, providing a transient and non-integrating alternative to conventional gene therapy while significantly reducing off-target expression
CM SMRTs allows for translation only in cardiomyocytes by leveraging endogenous microRNA (miR) profiles using two modRNA constructs. One encodes a suppressor (either L7Ae or Cas6) carrying CM-specific binding sites. The other encodes a target gene carrying the suppressor's recognition element (the K-motif or hairpin, respectively). Watch the video above to see it in action.
Our Regenerative Target: PKM2
Our latest work shows the success of using PKM2 CM-SMRTs to regenerate and revascularize juvenile and adult pig hearts post-MI, leading to reduced scarring and improved overall cardiac funcntion
From Bench to Bedside
Our preclinical successes have moved us rapidly towards first-in-human trials. We have demonstrated:
• First ever delivery of modRNA selectively into cardiac muscle
• Cardiac repair and prevention of heart failure in small and large animal models
• Precise, high-efficacy translation in small and large animal models
• Optimized lipid-nanoparticle formulations for cardiac delivery