CRISPR-Cas9 has been established as a viable means of editing DNA. However, the delivery of gene editing nucleases in-vivo remains a challenge as conventional vectors such as adeno-associated viri and protein carriers have poor serum stability, risky pathogenicity, nonspecific distribution, and off-targeting effects.
Moogene has developed and patented a biocompatible lipid nanoparticle technology platform to address these limitations; through methods such as antibody-mediated macropinocytosis and sonoporation, we have been able to administer gene editing proteins with therapeutic effect in indications such as KRAS-mutant cancer strains, androgenic alopecia, and type 2 diabetes. Our methodology shows precise and safe biodistribution, as well as high efficacy with no remarkable side effects such as off-targeting. We are aiming to conduct our first-in-human studies within a two-year time frame.