Addressing the challenges of using RNA as a therapeutic or a gene-editing tool

RNA as a therapeutic or a gene-editing tool

RNA technologies, including mRNA therapeutics, have emerged as a promising drug modality due to their essential biological role in protein expression and their potential versatility in manufacturing. As a result, RNA is in the global spotlight and is being prioritized due to the Covid-19 pandemic.

To maintain the current momentum as industry returns to a ‘normal’ cycle of drug and therapy development, BioPhorum experts have come together to identify and address the unique issues of the RNA modality. The result is a new paper, The challenges of using RNA as a therapeutic or a gene-editing tool.

An initial team of 38 subject matter experts from 15 companies identified dozens of challenges and grouped them into 15 distinct topic areas. These discussions were not intended to identify solutions but to outline what the BioPhorum team will be working towards in future activities. The high-level statements in this document will help focus subsequent discussions on delivering the detailed goals and benefits.

For each area, the paper summarizes the main problems, the goals of the further work and the benefits to companies and industry of addressing those problems.

The 15 topic areas are (not in order of priority):

  1. Defining terminology
  2. RNA DP route of administration, dosing strategy and distribution in a patient
  3. RNA pharmacodynamics and pharmacokinetics
  4. RNA-specific CQAs, CPPs and links to clinical performance
  5. RNA-specific assays and analytical tools/equipment
  6. RNA-specific reagents and raw materials and associated challenges, e.g., sourcing and use
  7. How does RNA compare against established therapeutic modalities?
  8. Operational requirements to establish an RNA platform/DP
  9. Regulatory guidance on RNA
  10. RNA advocacy groups
  11. CDMOs and CROs supporting RNA production/manufacturing
  12. mRNA stability, storage and handling, and shipment challenges
  13. Scale-up challenges for RNA DS and DP manufacturing
  14. Cleaning and safety considerations during the use of RNA and associated reagents
  15. Technical requirements and challenges for the use of RNA during manufacture.

The RNA team is now beginning to prioritize these topics, benchmark members’ experience and knowledge, and solve the associated challenges to establish and align best practices that will enable the promise of RNA to accelerate into a mature and well-established manufacturing and therapeutic platform. With recent significant progress and development in the field, now is a perfect time for industry and agency leaders to come together and discuss challenges and streamline the development of RNA technologies. This will maintain the current focus on this technology, both as a therapeutic and a component of the gene-editing toolbox.

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