Preparing for the Full Potential of RNA Therapeutics

Although several technical challenges remain, recent clinical and commercial successes of RNA-based therapeutics have demonstrated its potential as a modality. Now is the time for pharmaceutical companies focused on relevant areas—such as rare diseases, infectious diseases, and (potentially) oncology—to consider RNA-based therapeutics and their impact on the competitive landscape as part of their strategies. And other pharma companies need to evaluate when RNA-based therapeutics may become a relevant platform in their existing or emerging disease and therapeutic focus areas.

Some of the critical questions that pharma companies will have to ask themselves:

• What disease areas or type of indications are the current sweet spot for RNA therapeutics? How could ongoing technical advancements expand those and in what time frame?
• What role could RNA therapeutics play in your current focus disease areas? What are the relative advantages and disadvantages compared with your existing modalities?
• Given the above, should you participate in RNA-based therapeutics? If so, how and in what time frame?
• What are the appropriate signposts to reevaluate the role of RNA-based therapeutics or their threat to your current portfolio?
• How will you address the ongoing evaluation or incorporation of messenger RNA (mRNA) into your portfolio? What will be the resulting implications for capabilities and organizational requirements?

The answer to the above questions will evolve over time as the industry addresses challenges around stability, expression levels, and delivery efficiency of RNA therapeutics. We expect mRNA to compete more directly with small molecules, biologics, gene therapy, and gene editing—similar to what’s currently happening with vaccines and some rare diseases. Therefore, it’s imperative for pharma companies to understand the potential impact of this rapidly emerging area and evaluate the key strategic choices. Taking this critical action now can ensure that they aren’t blindsided down the road.