Shababa Masoud
Scientist Insitro
Seminars
Thursday 4th June 2026
Machine Learning Enabled ALS-Target Discovery & Development of an Oligonucleotide Therapeutic
11:30 am
- Discovery of novel genetic modifiers of TDP-43 dysfunction via machine learning-based screening in iPSC-derived motor neurons
- Multimodal data integration (POSH, Perturb-Seq) using an agentic framework enables selection of ALS-1 target
- In vitro target validation is shown through cryptic exon reversion, rescue of full-length transcripts, and functional improvement in neurite outgrowth
- Oligonucleotide-mediated target knockdown demonstrates potency and CNS distribution in vivo
- Detection of a putative target engagement biomarker in human cerebrospinal fluid
