There are currently no messenger RNA (mRNA) non-vaccine therapeutics on the market. However, this landscape will reach a total of five marketed products by 2028, generating over $2 billion in revenue across the seven major markets (7MM*), says GlobalData, a leading data and analytics company.

According to GlobalData’s recent report, ‘mRNA Therapeutics – Thematic Intelligence’, there has been a surge of interest in the mRNA therapeutics space following the COVID-19 pandemic wherein mRNA vaccines proved successful. mRNA is a single-stranded ribonucleic acid that is transcribed from a strand of DNA and carries the coding information for protein synthesis. mRNA therapeutics thus involve the delivery of in-vitro transcribed mRNA into a target cell in order to induce expression of a specific protein.

Pharma companies such as Moderna, Ultragenyx, Omega Therapeutics, and BioNTech are exploring non-vaccine mRNA therapeutics for cancer and rare genetic diseases. There are five candidates in clinical trial development under these four companies, all in Phase I/II. Their combined revenue is estimated to be upwards of $2 billion by 2028, with Ultragenyx’s OTX-2002 contributing most significantly to sales, generating $1.6 billion during 2028, according to analyst consensus forecasts on GlobalData’s proprietary platform, the Pharma Intelligence Centre.

Sarah Bundra, Pharmaceutical Analyst at GlobalData, comments: “mRNA therapeutics provide many unique benefits. Notably, they can act on targets that have previously been undruggable for small molecules and they provide individualized therapy by addressing specific protein deficiencies in a patient’s body.”

mRNA therapies also allow for the temporary expression of proteins, as opposed to altering a patient’s genetic makeup to incur permanent expression. mRNA therapies are administered to patients to compensate for defective or absent proteins. Therefore, this is an especially promising therapeutic option for patients with rare genetic diseases that are caused by genetic mutations, as their deficient or absent proteins can be transiently corrected through mRNA therapeutics. However, there are hurdles that need to be overcome in order to realize the full potential of mRNA therapeutics.

Bundra adds: “Delivery of mRNA in the body needs to be optimized, as mRNA molecules are susceptible to degradation by ribonucleases. This is a natural way that the body regulates gene expression but can be disadvantageous when dealing with mRNA therapeutics as it limits the drug’s in vivo efficacy. Additionally, introducing mRNA carries the risk of activating the immune system, which has to be mitigated for circumstances in which stimulating the immune system would be harmful for the patient; namely, when dealing with non-immunotherapeutic indications.”

*7MM: The US, 5EU (France, Germany, Italy, Spain, and the UK), and Japan