The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
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.
- Publication:
-
Nature Nanotechnology
- Pub Date:
- December 2019
- DOI:
- 10.1038/s41565-019-0591-y
- Bibcode:
- 2019NatNa..14.1084A