Describing AAV-Driven Safety Findings (Depatotoxicity & Peripheral Neuropathy) in the Non-Human Primate & Potential Translatability
Time: 9:30 am
day: Conference Day Two
Details:
- Outlining the future of preclinical development: Is in vivo modelling on the way out? Presentation of retrospectively analyzed data from preclinical non-human primate (NHP) studies to establish the time course of self-complementary AAV9(scAAV9)-related toxicology findings after systemic or intrathecal administration.
- Investigation of the potential triggers (capsid versus transgene) for such toxicities in the cynomolgus macaque, the pathological mechanisms at play and of the impact of immunomodulatory agents.
- Comparison/contrast between those nonclinical results with clinical findings.
