China-based Insilico Medicine announced the identification of multiple novel therapeutic targets for amyotrophic lateral sclerosis (ALS) using its AI-driven platform PandaOmics, in collaboration with Answer ALS. The findings, published in Frontiers in Aging Neuroscience on June 28, highlight 17 high-confidence and 11 new targets validated in ALS models.
Research Highlights
- AI Platform: PandaOmics analyzed CNS samples and iPSC-derived motor neurons from Answer ALS, the largest ALS research project.
- Target Identification: 17 high-confidence and 11 novel targets were discovered, with 18 (64%) validated in the c9ALS Drosophila model.
- Key Genes: Eight unreported genes (KCNB2, KCNS3, ADRA2B, NR3C1, P2RY14, PPP3CB, PTPRC, RARA) showed strong potential to rescue neurodegeneration.
ALS Context
ALS patients typically face a 2–5-year life expectancy post-diagnosis, with existing drugs failing to halt disease progression. Insilico’s findings aim to address this unmet need.-Fineline Info & Tech
