Redefining Preclinical Strategy: Leveraging Humanized Mouse Models to Accelerate Regulatory Pathways for CNS Gene Therapies
Time: 10:00 am
day: Conference Day One
Details:
- Highlighting engineered AAV capsids that effectively cross the blood-brain barrier by targeting human receptors
- Engineering capsids with specificity to a human receptor can render humanized mice highly effective for biodistribution, toxicity, and efficacy studies that are both translationally relevant and cost-efficient
- Streamlining to a single-species preclinical model reduces development time, ethical burden, and barriers to IND submission
