Integrating Human-Relevant Preclinical Models to De-Risk ASO Potency, PKPD Translation & Clinical Dose Selection in TDP-43–Driven Neurodegeneration
3:30 pm - Friday 21st August 2026
- Leveraging patient iPSC-derived neurons that recapitulate TDP-43 pathology to screen and rank splice-correcting ASO candidates by potency, efficacy, and safety-enabling identification of leads with clinically relevant characteristics
- Utilizing a humanized mouse model to establish in vivo proof-of-concept and generate PKPD relationships that bridge human cell-based potency to CNS tissue exposure and target engagement
- Presenting NHP PKPD and tolerability data to support translational dose selection and inform therapeutic index ahead of clinical entry
- Outlining a clinical development strategy informed by integrated preclinical evidence across human cellular, humanized rodent, and NHP platforms to enable efficient first-in-human study design
