siRNA-based Drugs



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siRNA-based Drugs

Since FDA approved first siRNA-based drug (patisiran) for polyneuropathy treatment, doors have been opened to utilize siRNA as a therapeutic agent for treatment of various diseases. Recently, FDA approved second siRNA drug (Givlaari) for rare disease. Moreover, there are at least 30 siRNA-based drugs in clinical trials process for several pathological conditions such as viral infections, cancer, genetic disorders, neurological disorders and autoimmune disorders. This demonstrates the significant potential of these therapeutic strategies, which promise to be effective in a wide range of currently untreatable disorders.

Compared with small molecular drug and protein-based drug, siRNA-based drug has distinct advantages, including high specificity, high potency and reduced toxicity. It also provides a strong option for those undruggable and untaggable targets of small molecules and antibodies. In addition, siRNA-based drug plays an important role in developing personalized anticancer therapeutics. Such personalized medicine is likely to be more effective in controlling the cancer, thereby improving the success rate of cancer drugs.

Table1. Comparison between small molecule drugs, protein-based drugs and siRNA-based drugs

PropertiesSmall molecularProtein-based drugssiRNA-based drugs
Nature of actionActivation or inhibitionActivation or inhibition targetsInhibition of targets
Site of target proteinExtracellular and intracellularMainly extracellularVirtually any sites
SelectivityVariableHighly specificHighly specific
Lead optimizationSlowSlowRapid

Although siRNA-based drugs are the latest frontier in nucleic acid therapeutics, with a considerable number in clinical trials. There are still significant barriers to efficient siRNA-based treatment strategies including delivery of the drug to a particular site or tissue, off-target effects and longevity of the treatment. Therefore, the future prospects of siRNA-based drugs will require improvements of delivery strategies to maximize drug efficacy while minimizing target toxicity and off-target effects.

As one of the global biotechnology companies specializing in RNA biology, IntegrateRNA provides custom siRNA synthesis, modification, screening services and products like siRNA libraries. Our siRNA services and products are used to knock-down a specific gene of interest for identifying gene function, elucidating pathways and screening for potential new drug targets to accelerate your drug development and clinical research. If you have any questions, please feel free to contact us.


  1. Kanasty R. et al. Delivery materials for siRNA therapeutics. Nature Materials, 2013, 12(11):967-977.
  2. Lieberman, Judy. et al. Tapping the RNA world for therapeutics. Nature Structural & Molecular Biology, 2018.
For research use only. Not intended for any clinical use.
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