CP0014753, a Potent, Selective & Well-Tolerated NAT8L-Targeting Antisense Oligonucleotide for Canavan Disease, Supported by Durable Modulation of Brain NAcetylaspartate in Non-Human Primates

  • Exploring PF/PD profiling of Contera’s lead ASO in non-human primates, including both juvenile and adult cohorts, with longitudinal measurements over 28 weeks to characterise tissue distribution, target engagement, and systemic exposure
  • Validating target engagement and compound selectivity using non-invasive proton spectroscopy biomarkers
  • Discover how extended preclinical evaluation in non-human primates informs translational development for establishing benchmarks for dosing, pharmacodynamic responses, and optimisation strategies for clinical readiness 

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