souseikai global clinical research center

Oligonucleotide Therapeutics

Oligonucleotide Therapeutics (ONTs) have been the center of attention in recent years as one of the promising therapeutic options that could meet the unmet needs of many therapeutic areas.

Since the first ONTs were approved in 2008, R&D has been moving ahead full throttle in Japan with active research and discussion among experts.

With the Japanese PMDA’s efforts supporting this new area, SOUSEIKAI has been proud to be a part of the trend, conducting safe early clinical trials for the last 3 years.

There are several kinds of ONTs, such as Antisense oligonucleotides, small interfering RNAs, microRNAs, Aptamers, Decoys, and CpG oligodeoxynucleotides. Our experience includes Antisense ONTs and Aptamers as of 2020, as well as other ONT study capabilities.

Approved Oligonucleotide Therapeutics in Japan (as of February, 2021)

Generic name Trade name Type Target Indication Year approved
Pegaptanib Macugen® Aptamer VEGF protein Neovascular (wet) age-related macular degeneration 2008
Nusinersen Spinraza® Antisense SMN2 pre m-RNA Spinal muscular atrophy 2017
Patisiran Onpattro® siRNA TTR mRNA Polyneuropathy of hereditary transthyretin-mediated amyloidosis 2019
Viltolarsen Viltespo Antisense Dystrophin pre-mRNA Duchenne muscular dystrophy 2020

Link: Considerations of the Japanese Research Working Group for the ICH S6 & Related Issues Regarding Nonclinical Safety Assessments of Oligonucleotide Therapeutics: Comparison with Those of Biopharmaceuticals