mRNA-based therapy meets EnPC®

Combining the EnPC® drug delivery technology with a mRNA-based transcript therapy approach provide for a new treatment option with regard to CNS-related genetic diseases with the following benefits:

• no risk of integration into the genome, which can lead to insertional mutagenesis with potentially disastrous effects. NEUWAY’s therapeutic mRNA results in transient translation that can be controlled by changes in the sequence and provides for a safer delivery approach compared to DNA-based therapeutic agents
• the mRNA does not access the nucleus after being delivered into the cell for translation
• mRNA based therapeutics do not need encoding of additional genes such as antibiotic resistance related genes
• packaging protects the mRNA from physical and chemical instability caused by extracellular ribonucleases and potentially immunogenic challenges of the load
• it ensures the passing and delivery of the mRNA through cellular membranes towards the cytoplasm

Rapid drug prototyping using EnPC technology