The following is an excerpt from the eBook RNA-based Therapies: Takeaways and Best Practices for Strategy Planning

Although no RNA therapies are yet approved in oncology, excitement is growing because novel RNA technologies that selectively inhibit and alter protein expression may offer immense long-term clinical benefits.

From immune-activating mRNA-based cancer vaccines to RNAi and aptamers that can selectively silence genes and inhibit pathways, these therapies are building significant momentum, fueled by the promise of great survival benefits.

Despite being a hotbed of optimism, the outstanding commercial potential for these novel therapies are mired by the unique technological challenges and high-risks associated with the lengthy clinical development remaining.

Here we outline 5 key takeaways for planning in this emerging market:

RNA market heating up despite risks: Development of RNA-based therapies remains highly dynamic given that clinical programs are predominantly in the early phases with a higher risk of failure. Despite this, the significant opportunity for growth has driven an increase in the number and size of drug developers.

Hard to treat indications with limited competition are best options for developers: Many of the ongoing early phase trials lack an active comparator; however, by focusing on hard to treat diseases with limited options, an agent that demonstrates eff­icacy is likely to appear promising, but more robust data will be needed to fully convince physicians and regulators of the benefits for this innovative class.

Relatively low manufacturing costs: Broadly speaking, the costs associated with the upscaling and manufacturing process for RNA-based therapies are far cheaper and simpler than the manufacture of other biological therapies.

Combination strategy should be explored, with caution: Combining RNA-based therapies with immune checkpoint inhibitors could significantly improve response rates, thereby leading to clinically meaningful benefits for cancer patients. While it is shrewd to investigate these agents in combinations with established therapies, the the contribution of all agents in the combination should be carefully considered especially given the increased risks of toxicity and higher costs.

Limited analogs available for new entrants: With no RNA-based therapies yet approved for oncology, new market entrants will have to rapidly learn from other indications and competitors when trying to navigate the regulatory landscapes.

Want more takeaways to help planning?

Read the full eBook for an analysis of the RNA based therapy landscape: 

  • Current leading therapies
  • Emerging players, agents and trials to watch
  • Disease management and access issues
  • Perspectives from oncologist KOLs
  • Takeaways for biopharma strategists

RNA-based Therapies: Takeaways and Best Practices for Strategy Planning


Author Bio

Josh Dawkins is a HCC, RCC, and NSCLC expert authoring report on unmet need, current treatment, access & reimbursement, and covering the disease landscape and market forecasts in 7 countries. Get in touch on Twitter or LinkedIn.

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