Sixty years ago, when messenger RNA (mRNA) was first discovered, it was understood to be a way of transmitting genetic information between DNA and ribosomes. After several key additional discoveries, RNA is now accepted as the key molecules linking nearly all biological pathways.
In 2017, surgeon Vinod Balachandran discovered T-cells in a tiny number of pancreatic cancer survivors’ blood which had developed the ability to identify, remember and fight back against proteins in the deadly tumours. After beginning research alongside the biotechnology company BioNTech and progressing to clinical trials testing mRNA vaccines in pancreatic cancer sufferers. Despite its progress, this work soon pivoted focus towards a much-needed coronavirus vaccination.
mRNA vaccines can protect against infectious diseases by teaching our cells how to make a protein, or a piece of a protein, that triggers an immune response inside our bodies. Now most well-known for its industry-defining role in the Pfizer-BioNTech and Moderna COVID-19 vaccinations, the success of these vaccinations has opened a world of opportunity for the treatment of other incurable diseases, including HIV and cancer.
Despite years of trials and research, until the rapid COVID-19 vaccine approval most mRNA-based products struggled to gain approval. Alongside BioNTech, other biotech giants such as Moderna and AstraZeneca are also studying the development of personalised cancer vaccines using mRNA. This progress could revolutionise cancer treatment, however the process still requires creation of individual vaccines tailored to specific cancers. As multiple patients with the same cancers do not necessarily have identical tumours, vaccines may even require development on a personal level to be fully effective.
It’s amazing to see how these breakthroughs have affected the wider market. The effect of huge companies exploring the possibility of cancer vaccinations using mRNA offers opportunities for up-and-coming biotech companies to gain real exposure too.
Companies like ExonanoRNA are developing RNA nanotechnology-based therapeutics for treating late-stage oncology diseases, mapped to join the market as soon as 2025. Through developing RNA Nanoparticles in a modular fashion, this method makes it easier to switch out specific modules and incorporate custom modalities. Allowing treatments to be tailored to the specific needs of the patient.
Skyhawk have its SkySTAR* (Skyhawk Small molecule Therapeutics for Alternative modulation of RNA) platform, which integrates information from computational, kinetic, and structural models of RNA to generate a unique formula for each target treatment. Its cancer target is currently in the pre-clinical stage, but with $133 Million secured in funding in September 2021 this looks to progress quickly in the coming year.
Bill Haney, co-founder and CEO of Skyhawk Therapeutics, said:
This investment round strengthens Skyhawk's capacity to advance our internal pipeline of drug candidates deep into the clinic.
Accent Therapeutics is also at the forefront of discovery in this space after being named one of ‘The Fierce 15’ biotech companies in 2020. Praised for championing innovation and creativity in the face of fierce competition, its cellular control mechanisms determine which RNA transcripts are translated into cell proteins and when. This new area of biology, collectively referred to as RNA-modifying proteins (RMPs), changes the chemical composition of RNA molecules to alter function or stability. Although the team at Accent is still working towards understanding the roles of RMPs in oncogenesis, a recent review highlighted a small sampling of RMPs and their reactions associated with human cancers as successful.
Rgenta Therapeutics is developing orally administered medicines, opposed to vaccinations. These small-molecule RNA-targeting treatments are initially focussed on oncology disorders. Rgenta’s patented platform mines masses of genomics data to identify targetable RNA processing events. It uses this to design small-molecule glues to modulate the interactions among the regulatory proteins and RNAs. This data-driven discovery platform accounts for cell type-specific target regulation, predicting the biological consequences of its use.
NextRNA is another company aiming to revolutionise treatment of ‘undruggable’ diseases such as cancer, this time using its platform to target the specific disease-causing non-coding RNA (ncNRA). Next is accelerating the development of therapeutics by specifically identifying proteins responsible for the ncRNA function and targeting these to disrupt further disease progression.Finally, Strand Therapeutics is planned to proceed with clinical trials in 2022. Its mRNA constructs use synthetic biology and mRNA therapeutics to combine genes for self-replication. Derived from RNA viruses genetically programmed to control location, timing, and intensity of therapeutic proteins within the patient’s body. This enables the precise and controlled delivery of multiple disease treatments in one single drug.
The novel coronavirus pandemic might have been the largest, most unpredictable human trial for RNA vaccinations in existence. Thanks to the fast forward in approvals required for its rollout, the future of oncology has changed for good. After huge companies like BioNTech started testing the waters, the innovative start-ups I’ve mentioned have made industry-defining moves to progress a vast range of RNA focussed oncology medicines. I’m really interested to see how these moves affect the future market and to understand if it’s truly possible to ‘drug the undruggable’.
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Small molecules are now able to target previously ‘undruggable’ conditions, such as cancers and neurological disorders, I discuss how we got here and what it means for the market. Click to read more.