mRNA therapy is based on the principle that a functional gene can also be delivered through mRNA an intermediate between DNA and protein. mRNA-based vaccines have revolutionised vaccine design and production, as seen during the COVID-19 pandemic. mRNAs are an alternate way to deliver good genes so that functional proteins can be made. The drug formulation is a non-viral, non-integrating mRNA-Liquid Nanoparticle formulation. It has two main components:
- mRNA Construct, that is, an RNA molecule that has all the information for the production of functional proteins.
- Delivery System, which allows RNA molecules to reach the desired cells without any damage. mRNA is encapsulated in lipid nanoparticles (LNPs) using a microfluidic mixing process.
TIGS has established the system, including mRNA design and synthesis and LNP formulation and production. They have shown expression of the GNE protein in muscle cells and in mouse muscle tissue. Currently, they are generating preclinical data for eventual submission to the drug controller. They are constructing a GMP (good manufacturing practice) facility to produce the formulation.