Journal
BIOTECHNOLOGY ADVANCES
Volume 37, Issue 5, Pages 801-825Publisher
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.biotechadv.2019.04.012
Keywords
ONPATTRO; Patisiran; siRNA; RNA interference; siRNA delivery; Chemical modification; Nucleic acid therapeutics; GalNAc; Liposome; Antisense oligonucleotide
Categories
Funding
- National Natural Science Foundation of China [31871003]
- Beijing Institute of Technology Research Fund Program for Young Scholars
- Fundamental Research Funds for the Central Universities
- Hunan Provincial Natural Science Foundation of China [2018JJ1019]
- Hu-Xiang Young Talent Program [2018RS3094]
- Postdoctoral Science Foundation of China [2018 M630085]
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Recently, United States Food and Drug Administration (FDA) and European Commission (EC) approved Alnylam Pharmaceuticals' RNA interference (RNAi) therapeutic, ONPATTRO (TM) (Patisiran), for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hAITR) amyloidosis in adults. This is the first RNAi therapeutic all over the world, as well as the first FDA-approved treatment for this indication. As a milestone event in RNAi pharmaceutical industry, it means, for the first time, people have broken through all development processes for RNAi drugs from research to clinic. With this achievement, RNAi approval may soar in the coming years. In this paper, we introduce the basic information of ONPATTRO and the properties of RNAi and nucleic acid therapeutics, update the clinical and preclinical development activities, review its complicated development history, summarize the key technologies of RNAi at early stage, and discuss the latest advances in delivery and modification technologies. It provides a comprehensive view and biotechnological insights of RNAi therapy for the broader audiences.
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