Delivering on the promise of RNA medicine

In September 2022, I wrote in Forbes about how advances in RNA medicines were driving the future of healthcare. While much has changed in the intervening years, my belief in the power of RNA medicines remains unwavering. In this short time, we’ve seen incredible progress across all aspects of RNA drug discovery, development and deployment but that progress can only be maintained and extended with continued research, investment and conviction. 

Since 2022, the field has made incredible strides towards realizing its potential:

On delivery and toxicity

While delivery of RNA to target cells remains one of the great challenges in genomic medicine, we’re making substantial headway. In particular, the improvement and optimization of lipid nanoparticles (LNPs) for delivery both in vivo and in vitro is clear. We’re extremely close to having programmable vehicles that can deliver therapeutic RNA molecules specifically to the disease-relevant cell types. This cell-type specificity can also increase the safety of these medicines. Scientists have also made strides in dialing up or dialing down the amount of therapeutic RNA made in the target cells, creating medicines that are tailor-made for particular diseases.  

On the synergy with gene editing

The first generation of gene editing applications delivered the editors, such as CRISPR, to target cells as complexes of proteins and guide RNAs. Increasingly, gene editors are being delivered to target cells as mRNA strands that are then manufactured by the target’s cellular machinery. This has enabled the advances in RNA medicines to be directly applied to gene editing applications and has supported the use of the next-generation of gene editors, including base and prime editors, which offer more precision and fewer off-target edits. We’re also seeing teams combine gene editing and RNA delivery with LNPs to make more sophisticated cell therapies.

On expanding indications

Since 2022, we’ve witnessed an impressive drum beat of results demonstrating the safety and efficacy of RNA medicines in an ever-increasing range of diseases. In pancreatic cancer and late-stage melanoma – both devastating diseases with low survival rates – mRNA-based immunotherapies have shown the ability to reduce the occurrence of relapse and increase response rates, respectively. Our immune systems are powerful, and RNA can be used to reprogram our immune cells to target the agents of disease, whether they be cancer cells, pathogens or the aberrant cells that cause autoimmunity. We’ve only just scratched the surface of RNA’s utility as a therapeutic modality.

At the crossroads

We’ve seen major increases in our ability to manufacture RNA and LNPs at scale, to design and test them rapidly and to optimize them across key parameters. Further integration of AI into these processes promises to speed our efforts to deliver these life-saving medicines to the patients who need them most.

Despite this wealth of progress, right now we’re standing at a crossroads. We can choose to step forward into this new era of medicine powered by RNA medicines—where cancer is a preventable disease, infectious diseases are contained, and we continue our efforts to help patients get the care they need and deserve—or we can step back, divesting from these programs just as they are poised to deliver results. 

I’m stepping forward, and I hope you will too.