About mRNA

mRNA as a Therapy

The current SARS Cov.2 pandemic has accelerated the use of messenger ribonucleic acid (mRNA) as a vaccine. mRNA is a single-stranded molecule, present in all of our cells. Identified in 1961, it carries instructions to cells for building proteins from our genes.

A segment of DNA is copied into a piece of mRNA that gives our cells commands for making specific proteins, some of which cause or prevent disease. The COVID-19 vaccines, for instance, contain the coronavirus’s mRNA (which does not cause infection). When the mRNA is injected into muscle, our cells read it and make the non-infectious spike protein found on the virus’s surface. This prompts our immune system to develop the tools to target and kill the virus if we then become infected later.

mRNA-based therapeutics have also shown great potential in the treatment of various other diseases. For instance, pilot studies have demonstrated that replacing the defective collagen VII protein is essential to improve the performance of wound healing in patients with recessive dystrophic epidermolysis bullosa.

Indeed, many rare genetic diseases are caused by dysfunctional or deficient proteins. Mutations in DNA can result in abnormal mRNA sequences, resulting in protein abnormalities. By providing the correct genetic information, mRNA therapies can regulate protein expression to improve or restore health.

As a result, protein replacement therapy has become a potential therapy for a wide array of rare monogenic diseases, blood disorders, and metabolic disorders such as AKU. The advantage of this technology is that mRNA delivery systems have been regarded as safe methods in disease therapies. The disadvantage is the requirement of multiple delivery because mRNA is not stable over a long period of time.

Our Technology

Our aim is to replace the defective mRNA which codes for the HGD enzyme in AKU patients.

To start, we will design and synthesize safe and effective mRNA constructs for replacement of the defective enzyme in AKU. We will evaluate the properties of mRNA-based therapies in pre-clinical models of AKU with a view to translate this into the clinic.

The biggest barrier to mRNA therapy has long been delivering mRNA to the correct place in the correct cells. Working with our partners, we will use nucleic acid delivery and specialized lipid-based formulations to target the liver and decrease HGA to normal concentration in serum.

We will also address the mode of delivery to avoid protein metabolism or clearance before entering the target cells and tissues. These include carriers such as nanoparticles, exosomes etc., which we will adopt to improve longevity of the mRNA.

Nanoparticle delivery vehicle encapsulating mRNA of desired protein enters the cell.

Desired protein produced is intracellular, transmembrane, or secreted.

An overview of mRNA-based therapy which we will target to the liver and kidney