As recently reported in Nature, the RNA interference (RNAi) drug patisiran has been approved by the FDA for the treatment of hereditary transthyretin amyloidosis (hATTR). hATTR is a genetic condition that is characterized by accumulation of misfolded transthyretin (TTR) protein in multiple organs and systems, including the nervous system. Patisiran works by targeting TTR mRNA to inhibit the production of mutant TTR, and is the first RNAi therapy to be approved by the FDA. The approval of patisiran represents the culmination of two decades of RNAi research, and is an important milestone not only for the treatment of rare disorders such as hATTR but also for RNAi therapy in general.
References
Original article
Ledford, H. Gene-silencing technology gets first drug approval after 20-year wait. Nature 560, 291–292 (2018)
Further reading
Fyfe, I. Treatment success in hereditary transthyretin amyloidosis. Nat. Rev. Neurol. https://doi.org/10.1038/s41582-018-0048-1 (2018)
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Wood, H. FDA approves patisiran to treat hereditary transthyretin amyloidosis. Nat Rev Neurol 14, 570 (2018). https://doi.org/10.1038/s41582-018-0065-0
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DOI: https://doi.org/10.1038/s41582-018-0065-0
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