TY - JOUR
T1 - RNAi therapeutic and its innovative biotechnological evolution
AU - Weng, Y.
AU - Xiao, Haihua
AU - Zhang, Jinchao
AU - Liang, Xing Jie
AU - Huang, Yuanyu
N1 - Publisher Copyright:
© 2019 Elsevier Inc.
PY - 2019/9/1
Y1 - 2019/9/1
N2 - 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.
AB - 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.
KW - Antisense oligonucleotide
KW - Chemical modification
KW - GalNAc
KW - Liposome
KW - Nucleic acid therapeutics
KW - ONPATTRO
KW - Patisiran
KW - RNA interference
KW - siRNA
KW - siRNA delivery
UR - http://www.scopus.com/inward/record.url?scp=85065515750&partnerID=8YFLogxK
U2 - 10.1016/j.biotechadv.2019.04.012
DO - 10.1016/j.biotechadv.2019.04.012
M3 - Review article
C2 - 31034960
AN - SCOPUS:85065515750
SN - 0734-9750
VL - 37
SP - 801
EP - 825
JO - Biotechnology Advances
JF - Biotechnology Advances
IS - 5
ER -