About Us
Non-viral gene insertion technologies represent the last significant missing gene therapy approach, offering the potential to treat diseases by introducing critical new or missing instructions to cells.
Averna Therapeutics is developing technology to safely and effectively deliver and durably integrate large sets of genetic instructions, something that viral approaches have struggled to do. Averna’s approach is based on RNA instructions that convert to stable DNA, delivered by lipid nanoparticles (LNPs). With our platform, we hope to expand the reach of genetic medicines, amplify the scope of addressable diseases, and broaden the patient populations able to benefit from this potentially life-changing class of therapies.
Our Approach
We are unlocking nature’s solution for precision gene insertion through the unique capabilities of site-specific retrotransposons.
Over a billion years of evolution, nature has developed a system that is primed to deliver on the promise of an RNA-LNP gene insertion system: retrotransposons. Of these, site-specific retrotransposons have evolved to insert at “safe harbor” genomic locations, which do not disrupt cell function. Averna is leveraging deep protein and RNA engineering expertise to adapt and modify one class of site-specific retrotransposon (R2) to reliably insert full genes of interest into safe harbors.
Our approach configures a modular, all-RNA system that is delivered by LNPs, which assembles in the cell to safely and efficiently insert any gene of interest. It can also be combined with multiple LNP delivery solutions.
