Public Funding for Vitarka Therapeutics Ltd

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.

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, Endo-Pore. Endo-Pore 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 robust _in vivo_ efficacy of our technology in delivering a clinically relevant therapeutic drug molecule into tumours. 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 UK.

Vitarka is developing a new tumour-targeted intracellular delivery technology for nucleic-acid therapies. Our project supports the UK life science strategy and the UK's industrial strategy to grow the bioeconomy and help patients. Existing intracellular delivery systems for nucleic-acid therapies predominantly depend on viral methods. These are toxic and lead to genome integration, causing adverse side effects such as cancer or death. This necessitates they must be administered in a centralised large-hospital setting with close patient monitoring. Therefore, there is need for a safe, easy to administer, non-viral intracellular delivery system. Our disruptive approach is to validate a new drug delivery technology, for intracellular delivery of into cancer cells, based on natural pore-forming proteins. This project fits within Fast Start because it is aiming to address the following problem: "_Self-driven healthcare, de-centralised healthcare and mental wellbeing through assessing new business models and routes to market for products and services related to self-care and self-management_" Vitarka is bringing forward an unprecedented therapeutic delivery technology with the potential to overcome safety limitations around previous technologies. This will enable much wider use of nucleic-acid therapy in oncology (e.g. non-small cell lung cancer, colorectal cancer, renal cell carcinoma and triple negative breast cancer), requiring much fewer hospital-based administrations of drugs than current medications and with the expected high safety margins that would mean a much lower level of monitoring and self-administration at home or under GP monitoring. This represents a cost-saving to the NHS as well as the opportunity for improved patient benefits. By validating this nucleic-acid therapy delivery system, Vitarka will enable safe administration of breakthrough therapeutics against hard to treat, lethal disease, effectively putting the UK at the forefront of scientific innovation and addressing urgent patient need.