The Role of mRNA
mRNA is a fundamental component of gene expression. It is the key link in the process of translating genetic information encoded in DNA into instructions that are used by cells to produce the proteins needed to carry out essential cellular functions. These instructions are processed through cellular mechanisms in two steps: transcription and translation. During transcription, a gene that encodes an amino acid sequence for a particular protein is transcribed into a complementary sequence of mRNA. The mRNA then carries these instructions to other areas of the cell where the instructions are translated by ribosomes, which are specialized molecular machines within cells that carry out protein synthesis. During translation, the ribosomes use the instructions conveyed by mRNA as a template for assembling the amino acids to create the desired protein.
mRNA and Disease
Abnormal gene expression, caused by a mutation in a DNA sequence, can result in the transcription of defective instructions. The translation of defective instructions by the cell can lead to the failure to produce, insufficient production or over production of a protein, or the production of dysfunctional proteins. This protein defect is the underlying cause of genetic disease.
To treat these types of disease, mRNA therapies have a potentially powerful ability to regulate protein expression and restore health.
Our mRNA Medicines
Translate Bio is developing transformative mRNA therapies to treat diseases caused by protein or gene dysfunction.
We believe that our MRTTM platform is broadly applicable across multiple diseases in which the production of a desirable protein can have a therapeutic effect. We are initially focused on restoring the expression of intracellular and transmembrane proteins, areas that have eluded conventional protein therapeutics, in patients with genetic diseases where there is high unmet medical need. We have observed successful production of desired proteins through multiple routes of administration in preclinical studies, which may allow us to develop MRTTM product candidates for the treatment of a wide range of rare and non-rare diseases.
Advantages of mRNA Therapeutics
mRNA therapy is engineered to deliver mRNA encoding natural, functional proteins that replace defective or missing proteins, and has potential advantages, including that it:
- restores gene expression without entering the cell nucleus or changing the genome;
- enables the treatment of diseases that were previously undruggable by using the cell’s own machinery to produce natural and fully functional proteins;
- has drug-like properties that are familiar to health care providers, including the potential to repeat and adjust dosing in a chronic setting; and
- permits rapid development from target gene selection to product candidate.
We are driven to pursue this technology because of its potential impact. mRNA’s central role in protein expression confers the potential for this approach to have broad applicability across multiple diseases in which the production of a desirable protein can have a therapeutic effect.