Restoring UNC13A Function in ALS: Translating In Vivo ASO Biology into Clinical Impact
- First in vivo evidence that ASO-mediated restoration of UNC13A cryptic exon function can modify disease-relevant biology, validating UNC13A as a therapeutically actionable target in ALS
- Why partial restoration of UNC13A may be sufficient to move the needle clinically, and how to think about biological sufficiency versus complete target correction
- The critical importance of early intervention, with data supporting delivery before irreversible neuronal loss to maximise therapeutic benefit
- The need for a companion biomarker strategy to guide patient selection and timing in clinical trials, ensuring the right patients receive treatment at the right stage