The unique macrophage-targeted delivery technology provides tremendous opportunity. Hence, RNATICS develops RNA therapeutics for a variety of inflammatory and fibrotic diseases with high unmet medical need.
The initial indication for RNATICS’ most advanced drug candidate, RCS-21, will be pulmonary hyperinflammation and fibrosis after viral infection such as influenza or SARS-CoV-2. RCS-21 specifically inhibits microRNA-21, which is among the strongest upregulated microRNAs in alveolar macrophages of individuals suffering from severe hyperinflammation after viral infection. Targeted inhibition of miR-21 has shown significant reduction of pulmonary inflammation, amelioration of lung damage elicited by scarring, and marked improvement of lung function in a therapeutic proof-of-concept study in a preclinical animal model of inflammatory lung damage.
Further inflammatory and fibrotic diseases of the lung are currently under investigation.
Formal safety and toxicology studies are currently ongoing to confirm the non-hazardous nature of the lead compound RCS-21. The Federal Institute for Drugs and Medical Devices (BfArM) has already given a positive assessment in a formal Advice Meeting as to the clinical development including design of Phase 1in healthy volunteers. The start of first-in-human clinical trials is planned for 2024.