Proactive Investors - Scientists Katalin Karikó and Drew Weissman were awarded a Nobel prize this week for a discovery that went under the radar for many years, until it was found to hold the key to stabilising mRNA vaccines used in the fight against the Covid-19 pandemic.
The researchers own the patents to the nucleoside modification technology licensed out to drug companies such as BioNTech (NASDAQ:BNTX), where Karikó once worked, which developed an mRNA vaccine against Covid-19 with Pfizer Inc (NYSE:PFE), and Moderna (NASDAQ:MRNA).
A ‘messenger’ ribonucleic acid, or mRNA, vaccine works by delivering a segment of the mRNA found in proteins located on a virus’s outer membrane into the bloodstream to encourage an immune response that can fight the disease.
RNA holds the genetic code in many viruses, instead of another nucleic acid DNA (deoxiribonucleic acid), acting as a precursor to make viral proteins.
The Nobel prize committee praised Karikó for remaining true to her vision of using mRNA as a therapeutic despite the difficulties she faced convincing funders of its significance.
Karikó, a Hungarian-born scientist, began her work investigating the use of ‘messenger’ ribonucleic acid as a therapeutic while she was an assistant professor at the University of Pennsylvania in the early 1990s.
There she later met immunologist Weissman, with whom she collaborated on research to discover how RNA interacts with the immune system.
Their therapeutic breakthrough was to discover that modifications to the nucleoside bases, or building blocks, in RNA, could prevent a potentially fatal inflammatory reaction.
This allowed for the development of safe vaccines against Covid-19 that incorporated base-modified mRNA, with efficacy rates of about 95%.
The Nobel-winning discovery was key to modifying RNA so it remained stable enough to produce an effective vaccine, and it has been highly effective in curbing the spread of the respiratory disease.
A study by The Commonwealth Fund estimated that Covid-19 vaccines have saved 3.2 million lives through to November 2022 in the United States alone.
That same technology might one day be used to treat cancers, as many pharmaceutical companies have diversified into oncology in the wake of Covid-19, with a surge in cancer vaccine trials in 2021.
Europe, where Moderna’s expansion is expected to lead to an acceleration of the industry, is the second-biggest market for mRNA therapeutics after the United States.
Valued at a combined US$34.7 billion in 2022, the market is expected grow to about US$68.1 billion by 2030, expanding at a compound annual growth rate of between 8.8% and 13.2%, according to estimates by Extrapolate and Precedence Research.
What other mRNA technologies may have been overlooked?
Major consolidated players in the mRNA therapeutics market include Moderna Therapeutics Inc, Pfizer Inc, Merck & Co Inc, Sanofi (EPA:SASY) (which bought mRNA therapeutics company Translate Bio in 2021), BioNTech SE (NASDAQ:BNTX), GSK PLC (LON:GSK), Novartis (LON:0QLR), and AstraZeneca PLC (NASDAQ:AZN).
All these companies could benefit from the increased attention on mRNA and related vaccines.
Here are some other stocks lesser known in the pharmaceutical space that could be worth looking at.
Crispr Therapeutics
Based in Switzerland, CRISPR Therapeutics AG was one of the first companies to use CRISPR gene-editing technology developed by Nobel laureate Jennifer Doudna and the company’s co-founder Emmanuelle Charpentier to treat diseases.
It specialises in gene editing and gene therapies, including RNA-based technologies, with a specialisation in sickle cell anaemia.
Crispr Therapeutics' London-listed shares are up by about 5% for the year to date at a price per share of about US$42.80.
Noxopharm
Australia-listed Noxopharm Ltd (ASX:NOX) is ahead of the pack because it diversified its therapeutic offering in the wake of Covid-19 pandemic into cancer treatment.
The US Food and Drug Administration (FDA) granted orphan drug designation status to a pre-clinical drug for the treatment of pancreatic cancer earlier this month, which has a poor five-year survival rate of just 9%.
Its shares have risen 187% this week to A$0.12.
Croda International
Croda International PLC (LON:CRDA) was identified by analysts at investment bank Berenberg in July 2021 as having a potential opportunity in mRNA vaccine growth, though reported its customers were destocking Covid-19-related products earlier this year.
Through Avanti, a company it bought in August 2020, it supplies lipids used in mRNA inhalation-based therapeutics, as well as offering crop-enhancing ingredients.
Its share price has risen steadily since inception and its shares are currently trading up at about 4,783p per share, but have yet to recover to the heights of late 2021 when it peaked during the Covid-19 crisis.
Meanwhile, Nasdaq-listed RNA medicine and vaccine company Arcturus Therapeutics, which has an mRNA manufacturing tie-up in Japan with Arcalis, has seen its share price climb by more than 50% this year.
Other notable players operating in the mRNA market include London Stock Exchange-listed gene therapy company Sangamo Biosciences, Inc., scientific data company San Francisco-based Benchling, Massachusetts-based RNA engineer Orna Therapeutics, Japanese healthcare company Daiichi Sankyo Co. Ltd., and CureVac NV.