The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therapies enabled by nanoparticle delivery.
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A.A., M.A.M., M.M., K.F., M.J. and S.B. are employees of Alnylam Pharmaceuticals. S.A., X.D., M.J.H., T.D.M., B.L.M., S.C.S. and Y.K.T. are employees of Acuitas. P.R.C. has financial holdings in Acuitas.
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Akinc, A., Maier, M.A., Manoharan, M. et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat. Nanotechnol. 14, 1084–1087 (2019). https://doi.org/10.1038/s41565-019-0591-y
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DOI: https://doi.org/10.1038/s41565-019-0591-y
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