A CRISPR PLATFORM THAT DELIVERS
Through a biologic approach, our uniquely differentiated proprietary technology resolves the greatest unmet need in gene editing: cellular delivery to develop groundbreaking therapeutics in oncology.
THE PROBLEM WE SOLVE
Current gene editing systems use adeno-associated viruses, liposomes or other nanoparticles to shuttle the nuclease to cells. These methods are limited by the types of targets/tissues they can reach, their safety profile and complex/costly manufacturing. Our platform directly delivers in vivo the safest and most efficient form of CRISPR : the ribonucleoprotein. No viruses, lipids or nanoparticles needed.
Featuring a plug and play system and a rapid iterative lead selection process, our platform exquisitely tailors cellular delivery for a largely extended spectrum of targets.
SYSTEMIC, TARGETED IN VIVO DELIVERY
Our platform produces tailored gene editing complexes with specific affinity to a variety of different cells. The therapy is deliverable intravenously while achieving highly targeted in vivo edits.
A FULL SPECTRUM OF POSSIBILITIES
Multivalence and unprecedented frequencies of homology-directed repair allow the performance of disruptions (knock-outs), corrections (knock-ins) and complex genetic manipulations significantly widening the range of possible therapeutic strategies and interventions.
SAFETY AND HIGH EFFICACY
The direct delivery of the ribonucleoprotein presents the optimal safety profile in terms of off-target incidence and immunogenicity. In clinically relevant models in-vivo, our gene editing complexes showed high efficacy and tolerance, excellent bio distribution and safety from off-targets and immunogenicity.
The production of our gene editing complexes follow the same well established manufacturing processes as biologics, ensuring high quality manufacturing standards, short production times and significantly reduced cost of therapies.