What’s Next in mRNA After COVID-19?
The COVID-19 pandemic brought its fair share of disruption and uncertainty; propelling biopharma-related topics into the public consciousness. The development of mRNA vaccines took centre stage. Investigated as potential treatments since the 1990s, companies like Moderna and Pfizer/BioNTech gave them top billing. Though their time in the limelight was relatively short, the role that mRNA-based vaccines played in saving lives and nurturing the world back to normal has been widely acknowledged.
So, what’s next? The broad applicability of mRNA in making vaccines and treatments has meant the industry has not lost its enthusiasm for using it to prevent and treat rare and not so rare diseases, including some cancers and viruses such as HIV.
As a company focusing solely on mRNA vaccine technology, the pandemic provided Moderna the impetus to build on its expertise. Although seasonal COVID-19 boosters will stay for the foreseeable future, Moderna has a vast mRNA pipeline focusing on a wide range of modalities including prophylactic vaccines, localized regenerative therapies and inhaled pulmonary therapeutics.
Pfizer are focusing on partnerships to deliver a similarly wide range of mRNA vaccines and treatments. Building on their success with BioNTech, a partnership that started in 2018, the partners are advancing studies on vaccines to treat both shingles and flu. And in an exclusive research partnership with Beam Therapeutics, both companies are focusing on in vivo editing programs aimed at several rare genetic diseases of the liver, muscles, and central nervous system.
Separately, partnerships with Acuitas Therapeutics and Telesis Bio seek to advance the overall effectiveness of mRNA vaccines through nanoparticle delivery systems and enzymatic DNA synthesis technology, respectively. An experimental mRNA therapy was recently delivered to cancer patients in the UK. The aim of the phase 1/2 trial, run by Imperial College Healthcare NHS Trust, was to assess the therapy in treating melanoma, lung and other ‘solid tumour’ cancers.
And moving on from mRNA, there is now interest in developing saRNA vaccines and therapeutics. In simplistic terms, small activating ribonucleic acid (saRNA) vaccines include a built-in ‘photocopier’ that allows for lower doses (1/64th) than mRNA vaccines whilst achieving equivalent levels of protection. The self-amplifying component of saRNA vaccines ensures RNA continually copies itself and persists within the body for longer - ensuring greater efficacy. Demonstrating its effectiveness versus traditional mRNA vaccines, Japan recently approved an saRNA COVID-19 vaccine after it demonstrated favourable results over its mRNA comparator.
Looking further, utilizing mRNA that encodes CRISPR-Cas 9 gene-editing proteins could offer single-dose treatments as a viable solution by targeting genes that are known to cause specific diseases. MRNA-based genome editing may be safer and more efficacious for patients, and holds the dual promise of lower costs and wider flexibility in delivery methods from a manufacturing perspective.
