Developing non-viral nanopore drug delivery technology for first-in-human trials

A new wave of RNA-therapeutics has the potential to transform outcomes for patients with cancers which do not respond to existing small molecule or antibody treatments. RNA-therapeutics have come to the forefront on the back of Covid vaccine success, and many Biotechs are now developing these for currently non-responding patient groups. This £6Bn potential market of new RNA-therapeutics needs a safe drug-delivery technology: to protect the therapeutics from degradation within the body, to target them to the tumour, and to release them inside the cells.

Vitarka is a UK life sciences technology SME which is developing a novel intracellular delivery technology, EndoPore. EndoPore is a drug delivery technology which will carry RNA-therapeutics through the body and then release them within tumour cells - opening up many currently validated but undruggable targets for new anti-cancer treatments.

Through this grant, we will substantially de-risk the development path by establishing formulation and GMP-grade large-scale manufacturing of our technology for first-in-human clinical trials. We will start validating clinically relevant formulations and define the roadmap for regulatory interaction. This will position the programme for larger scale private investment, and for partnership with biotechs for clinical trials, thereby enabling rapid technological and commercial impact for Vitarka and the UK.

We have successfully developed end-to-end process for large-scale manufacturing, and we have optimised upstream and downstream processes, which can be transferred to GMP grade manufacturing. Over the course of development of large-scale manufacturing method, it has become evident that the purification of insoluble and untagged proteins leads to significant reduction of pure protein yield. As we require additional quantities of proteins to perform formulation and characterisation studies, we will now outsource production of these proteins to a subcontractor enabling Vitarka to complete the project in preparation for first in human trials of EndoPore delivered therapeutic.