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Drastic advances have rapidly broadened the therapeutic potential of RNA therapeutics. Previously limited to vaccines targeting infectious diseases, RNA therapeutics in the development pipeline are now also targeting various cancers, rare diseases, and neurological diseases [1]. Breakthrough RNA therapies are also making precise genome and epigenome editing a reality, enabling greater control of genes responsible for disease.

Despite the possible benefits these therapeutics could bring to patients, many challenges are hindering their delivery to clinical trials and beyond.

Obstacles on the path to RNA therapeutic success

Realising the promise of breakthrough RNA therapeutics relies on drug developers overcoming several obstacles:

  • Challenging development and manufacturing processes
    RNA therapy production is a multifaceted and intricate process that demands a comprehensive understanding of not only the development and manufacturing of nucleic acids but also of plasmids and lipid nanoparticles (LNPs).
  • Navigating a complex and changing therapeutic landscape
    Although RNAs as therapies is not a new concept, the success of these therapeutics is still relatively new, there are currently no established guidelines for their production. However, the RNA space is quickly evolving, with regulatory bodies responding to new advancements and innovations. Developers and manufacturers must stay ahead of the curve to meet changing regulatory requirements, now and in the future.
  • Responding to timeline pressures
    RNA developers are under pressure to help patients faster and demonstrate the potential for a return on investment. As a result, developers face the challenge of delivering their innovative therapies to critical milestones within tight timelines.  

Propelling breakthrough RNA therapies

Conquering these challenges relies on strategies that allow developers to be flexible, altering processes to meet changing needs as the project progresses.

In our most recent infographic, Jing Zhu, Vice President, Nucleic Acid & Viral Vector Technology and Melanie Cerullo, Chief Quality & Regulatory Officer at ReciBioPharm explore the factors needed to ensure the smooth delivery of RNA projects from preclinical to commercial.

Discover how we’re progressing breakthrough RNA therapies with inspired science.

Read more

References:

  1. American Society of Gene+Cell Therapy. Gene, Cell, + RNA Therapy Landscape Report. Q4 2023 Quarterly Data Report.