Jorge Santiago-Ortiz

Company: Apertura Gene Therapy

Job title: Senior Director, CMC

Seminars:

Redefining Preclinical Strategy: Leveraging Humanized Mouse Models to Accelerate Regulatory Pathways for CNS Gene Therapies 10:00 am

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…Read more

day: Conference Day One