神戸にて開催される日本核酸医薬学会第10回年会に出展いたします。ランチョンセミナーでも発表いたしますので、ぜひお気軽にお立ち寄りくださいませ。7月2日（当日） 8時より整理券を配布いたします。
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小間番号：25
ランチョンセミナー
日時：7月2日 12:55 ~ 13:55
場所：3階 3B会議室（ランチョンセミナー2-4）
発表タイトル：An integrated biology platform to support discovery of oligonucleotide drugs
講演者：○Xiaoting Du, Zenghui Huang, Shibo He, Ya Sun, Qiugang Lei, Junmei Zhang, Qiong Zhou
要旨：
Oligonucleotide therapies such as antisense oligonucleotides and small interfering RNAs are a transformative class of drugs, with over 18 approved therapies for a genetic, metabolic and rare diseases. Positioned to become the third major drug modality after small molecules and biologics, these agents modulate disease pathways at the RNA level. However, their development faces persistent challenges such as efficacy, delivery, and toxicity including off-target effects.
To address these bottlenecks, we have established an integrated oligonucleotide drug discovery platform including sequence design, chemical synthesis, in vitro/in vivo activity profiling, early toxicity screening, and delivery system discovery and evaluation. Leveraging machine learning technology, we established proprietary design pipelines that prioritize sequences by drug-like potential. Our tiered screening funnel combines high-throughput cellular assays, PK/PD in normal or humanized mouse models, as well as efficacy studies in rodent and non-human primate disease models. To address delivery challenges, we established a range of assays including binding, internalization, and endosome release assays to evaluate and optimize delivery tools. Additionally, off-target assessment via RNA profiling, immunogenicity, and other early toxicity tests are incorporated into the screening cascade to reduce drug safety risks.