mRNA Platform

Why mRNA?

01
Direct Approach
Enables replacement of large proteins
02
Targeted
Preclinical evidence of preferential targeting of specific muscle cells with systemic injections
03
Repeat Injections
Adaptable for treatment across range of patients

Messenger RNA (mRNA) is a single-stranded RNA molecule that is complementary to one of the DNA strands of a gene. The mRNA is an RNA version of the gene that leaves the cell nucleus and moves to the cytoplasm where proteins are made. During protein synthesis, an organelle called a ribosome moves along the mRNA, reads its base sequence, and uses the genetic code to translate each three-base triplet, or codon, into its corresponding amino acid.

mRNA can be used as protein replacement therapy to treat diseases caused by an absence of protein, or by defective proteins, such as Cystic Fibrosis. If a gene has a mutation that stops it from producing protein or causes it to produce defective protein, mRNA medicine can provide a healthy version of the missing protein.

To function in vivo, mRNA requires safe, effective and stable delivery systems that protect the nucleic acid from degradation and allow cellular uptake and mRNA release. Initial techniques used naked RNA, which is prone to RNase degradation and evokes a strong proinflammatory response. More sophisticated methods sought to enable cell entry and, on a systemic level, to allow sufficient circulation time for the therapeutic mRNA to reach its destination and be released into target cells.

Lipid Nanoparticles have successfully entered the clinic for the delivery of mRNA; in particular, Lipid Nanoparticle–mRNA vaccines are in clinical use against the coronavirus disease since 2019 (COVID-19), which marks a milestone for mRNA therapeutics. To date, Lipid based Nanoparticles (LNPs) are the only RNA therapeutic carriers.

Our mRNA technologies:

The structure of the mRNA affects it’s performance. This includes potential immunogenicity, efficacy of translation and stability in the patient. We design, synthesize, and formulate our therapeutic mRNA, to suit the desired application.

mRNA Platform