RNA therapies by therapeutic class

Beacon RNA tracks 1327 RNA-based therapies which treat both cancer and non-cancer indications. These therapies are categorized into 5 main therapeutic classes: oligonucleotides, ribozymes, mRNA, guide RNA (gRNA)-mediated gene editing and RNA-targeted small molecules.

RNA trials by phase and status

  • Beacon RNA currently tracks 1029 trials investigating RNA therapeutics across a range of oncology and non-oncology indications, the most common being COVID-19 (12%), Duchenne muscular dystrophy (5%) and hepatitis B (4%).
  • The trial landscape is continuing to develop with 62% of completed and active trials in phase 1 and phase 2, indicating growth and investment in the RNA space as more drugs transition from preclinical development into the clinic.
  • In line with the 21% compound annual growth rate in initiation of trials, we expect that many more RNA-based therapies will enter the clinic in the coming years.

Key Learnings

While the current therapeutic class landscape significantly favors oligonucleotide-based, mRNA-based and gRNA-mediated gene editing therapies, which together account for 90% of all RNA-based therapies, the number of RNA-targeted small molecules is increasing, and we expect to see increased activity in this space.

The organizations on Beacon RNA range from small biotech startups dedicated to a single RNA modality to big pharma which have been established in the RNA field for years. The range of developers has given rise to a diverse portfolio of RNA therapies which use a variety of RNA platforms and technologies such as ADAR.

Triggered by advances in research and 18 drug approvals, RNA therapies are experiencing a surge of activity and investment.

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This insight and data is part of the subscription to Beacon RNA.


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About Beacon Targeted Therapies

We are a clinical trial and pipeline database solution, designed in partnership with pharmaceutical professionals. We track targeted therapies to provide accurate, in-depth and real-time information in the rapidly evolving landscape of drug development.


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