Gene 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. mRNA delivered therapeutically only 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.
  • delivered mRNA does not access the nucleus and only needs to reach the cytoplasm for translation being particularly beneficial for non-dividing target cells in the CNS.
  • mRNA based therapeutics do not need encoding of additional genes such as antibiotic resistance related genes.
  • protects packed mRNA and thereby physical and chemical instability of the mRNA caused by extracellular ribonucleases and potentially immunogenic challenges of the load
  • ensures passing and delivering packed mRNA through cellular membrane towards the cytoplasm.

Rapid drug prototyping using EnPC technology