The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

Akin Akinc, Martin A. Maier, Muthiah Manoharan, Kevin Fitzgerald, Muthusamy Jayaraman, Scott Baros, Steven Ansell, Xinyao Du, Michel J. Hope, Thomas D. Madden, Barbara L. Mui, Sean C. Semple, Ying K. Tam, Marco A. Ciufolini, Dominik Witzigmann, Jayesh A. Kulkarni, Roy van der Meel, Pieter R. Cullis (Corresponding author)

Research output: Contribution to journalComment/Letter to the editorAcademicpeer-review

28 Citations (Scopus)

Abstract

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.
Original languageEnglish
Pages (from-to)1084-1087
Number of pages4
JournalNature Nanotechnology
Volume14
Issue number12
DOIs
Publication statusPublished - 4 Dec 2019

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    Akinc, A., Maier, M. A., Manoharan, M., Fitzgerald, K., Jayaraman, M., Baros, S., Ansell, S., Du, X., Hope, M. J., Madden, T. D., Mui, B. L., Semple, S. C., Tam, Y. K., Ciufolini, M. A., Witzigmann, D., Kulkarni, J. A., van der Meel, R., & Cullis, P. R. (2019). The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nature Nanotechnology, 14(12), 1084-1087. https://doi.org/10.1038/s41565-019-0591-y