RNA-based Drug Discovery



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RNA-based Drug Discovery

Cellular RNAs play crucial roles in cellular information transfer, gene regulation and disease progression. As the FDA-approved RNA-based drugs and prophylactic vaccines start to enter clinical medicine, this new class of biologics is poised to transform drug discovery and spearhead the transition to an information-guided drug delivery paradigm. Globally, there are 160 companies and 65 academic teams currently involved in RNA-based drug discovery and therapy. This implies that RNA-based drugs have become more promising as potential therapeutics in comparison to DNA-based drugs.

There are on the verge of an era of new drugs that tap into RNA biology. Most of these drugs use nucleic acid analogs and take advantage of complementary base-pairing to mimic or antagonize endogenous RNA processes. Now, RNA-based drug discovery has primarily focused on four modalities:

  • mRNA vaccines for cancer and infectious disease.
  • In vitro transcribed (IVT) mRNAs to replace or supplement proteins.
  • Antisense oligonucleotide, or RNA interference (RNAi) via miRNAs and siRNAs, to partially or completely turn off gene expression.
  • RNA aptamers, or 'chemical antibodies', which bind to specific molecular targets and can act as drug carriers to deliver small-molecule chemotherapeutics, siRNAs, miRNAs or nanoparticles into targeted tissues.

The first notable success for RNA-based drug was the FDA approval of the RNA aptamer, Macugen, for the treatment of AMD in December 2004. Since then, two antisense RNA drugs (Exondys 51 and Spinraza) and two siRNA drugs (Onpattro and Givosiran) were approved by FDA for the treatment of duchenne muscular dystrophy, spinal muscular atrophy, hATTR amyloidosis and acute hepatic porphyria, respectively.

Advantages of RNA-based drugs

  • Active on 'undruggable' targets
  • Easy and rapid design
  • Chemical synthesis without the variability of biologics
  • Cost effective
  • Easy to combine into drug cocktails, providing flexibility for personalized drugs
  • Low immunogenicity

These advantages of RNA-based drugs boost the translation into various clinical applications, from repairing genetic disorders to fighting against cancers to silencing virus replication. However, there currently exist two major challenges in RNA-based drugs: instability of RNA drugs in physiological environment and difficulty in delivery of RNA drugs. These can be resolved by chemical modifications of RNA and the improvements of delivery strategies.

RNA-based Drug DiscoveryFigure 1. RNA-based drug delivery strategies.

Significant intellectual and innovative input is required from experienced scientists across a variety of disciplines and backgrounds to discover a quality drug candidate. IntegrateRNA focuses on RNA biology to offer customers comprehensive solutions, services and products. Our client-focused, collaborative approach creates true partnerships that anticipate challenges, overcome obstacles and move us forward together on the journey of getting new drugs to market.


  1. Lieberman, J. et al. Tapping the RNA world for therapeutics. Nature Structural & Molecular Biology, 2018.
  2. Kenneth L. et al. Latest development on RNA-based drugs and vaccines. Future Science OA, 2018, 4(5):
  3. Ingmar H. et al. RNA-based drugs and vaccines will launch a new era of information-driven medicine. Drug Discovery, 2015.
For research use only. Not intended for any clinical use.
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