Abstract：
:Recently, United States Food and Drug Administration (FDA) and European Commission (EC) approved Alnylam Pharmaceuticals' RNA interference (RNAi) therapeutic, ONPATTRO™ (Patisiran), for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) 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.
journal_name
Biotechnol Advjournal_title
Biotechnology advancesauthors
Weng Y,Xiao H,Zhang J,Liang XJ,Huang Ydoi
10.1016/j.biotechadv.2019.04.012subject
Has Abstractpub_date
2019-01-01 00:00:00pages
801-825issue
5eissn
0734-9750issn
1873-1899pii
S0734-9750(19)30071-0journal_volume
37pub_type
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journal_title：Biotechnology advances
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