RNA-based therapeutics have transformative potential in medicine. However, the limited delivery technologies available to turn these molecules into effective drugs hold back their true power. Procavea Biotech has developed an innovative solution to this problem: a novel RNA delivery platform based on nonviral protein cages. When compared to approved delivery modalities and those in development, our technology has distinct advantages in its stability, engineerability, and ease of manufacturing. Although a handful of siRNA-based therapies have been approved with many more in the pipeline, there are still two major hurdles to be overcome. Firstly, the currently approved siRNA delivery systems are only suitable for liver delivery, with extrahepatic tissues remaining an important challenge. Secondly, of a given dose, less than 1% of the siRNA reaches its biological target due to endosomal entrapment. We have developed an innovative delivery system with the potential to address both of these critical limitations and bring siRNA-based therapies to yet unreachable disease targets.

We have established the ‘OP’ delivery system, which is the first non-viral protein cage specifically designed for the delivery of oligonucleotides to cells. At the beginning of the project, we had demonstrated the in vitro delivery of small molecule drugs and oligonucleotides. Through the Innobooster, we have generated in vivo proof-of-concept data showing the both the delivery of siRNA to extrahepatic tissues in a living animal, and, importantly, effective knockdown of a target gene. This milestone represents an important breakthrough in the validation of the OP platform. As we move forward, our focus is on pre-clinical development of a first siRNA-based drug candidate for a disease indication that is unreachable with other delivery technologies, with the aim of making a tangible impact for patients with poor, or even no, treatment options.