Rare and complex genetic diseases lead to significant disability, death and associated cost burdens for society. Complex genetic diseases include cardiovascular diseases and cancer, the two leading causes of death in the EU, and rare genetic diseases, which affect up to 36 million Europeans. Nucleic acid therapeutics represent a potential treatment, but they are hampered by the “delivery problem.” Systemically administered naked RNA is quickly degraded by endonucleases, can provoke adverse immune reactions and has off-target toxicity. Viral and lipid nanoparticle delivery vectors have toxic side-effects and are difficult to produce and target to specific tissues.
TraffikGene-Tx’s delivery platform is an alternative based on small peptide carriers that can be easily conjugated with hydrophobic tails. The peptides used are safe, as they are biodegradable, non-immunogenic and are rapidly cleaved in cells, avoiding the detergent effect of lipid nanoparticle components. In contrast to classical lipid nanoparticles, which typically accumulate in the liver, these compounds have shown targeting to other organs such as spleen, with excellent delivery to the cytosol. Their production is simple, cheap, and can be automated. The modular design and the possibility for high-throughput screening allows the collection of big amounts of data that can be used to develop a predictive model and facilitate the design of future carriers with the optimal delivery properties for a given function, accelerating the development of new carriers.
TraffikGene-Tx capitalises on the current breakthroughs in healthcare represented by nucleic acid therapeutics. RNA-based therapeutics are now emerging driven by the success of mRNA vaccines and TraffikGene-Tx aims to deliver them to the tissues where they are needed, breaking new ground in the field of RNA. Our objective is to validate our delivery vehicles in industrially and regulatory relevant contexts and start developing our pipeline to advance to the preclinical trial stage with therapeutic RNAs, with the goal of launching a company that will develop both and internal and external therapeutic pipelines. Our products will contribute to bring many promising nucleic acid therapeutics to the market, and help millions of patients, while reducing health care costs.
