Kylin Therapeutics, Inc. takes advantage of the RNA Nanotechnology platform called ‘packaging’ RNA (pRNA) inspired by the highly evolved design of a molecular motor found in the phi29 bacteriophage. pRNA exists in individual subunits, made up of only RNA, that self-assemble under ambient conditions into dimers, trimers, quatramers, and hexamers. Promising RNA-based therapeutic modalities including aptamers, ribozymes, and therapeutic RNAs have been incorporated into individual subunits of pRNA. All RNA-based therapeutics maintain both structure and function when incorporated into the pRNA system. Combining pRNA and therapeutic RNA molecules will revolutionize the development of targeted RNA therapeutics.

pRNA complexes can be used to deliver RNA-based therapeutics specifically into diseased cells. Targeting molecules (i.e. small molecules like folate, peptides, protein-binding aptamers, and monoclonal antibodies) can be easily incorporated into individual pRNA subunits. Once incorporated, pRNA is efficiently taken up by diseased cells via the targeting mechanism. After the diseased cells take up the nanoparticle, therapeutic RNAs are cleaved. Once released the released RNAs silence the genes that are causing the disease. Targeting mechanisms and gene silencing capabilities can be tailored to match the appropriate diseased tissue making the potential for the pRNA nanotechnology platform nearly endless.

Kylin is currently developing a robust pipeline of novel pRNA-based Nanotechnologies to treat multiple types of cancer. We are undergoing pre-clinical studies to determine in vivo toxicity, specificity, pharmacokinetics, efficacy, and biodistribution of the initial products in the Kylin pipeline.