Early Toxicology Studies for AAV Gene Therapy to Treat Friedreich’s Ataxia
Time: 2:00 pm
day: Day Two Whole
Details:
- Findings from preclinical toxicology studies evaluating the safety profile of AAV-based gene therapy for Friedreich’s Ataxia
- Leveraging preclinical findings to guide dose selection, identify potential safety concerns, and optimize treatment strategies for human patients
- Explore critical data on potential vector-related toxicities, biodistribution patterns, and long-term effects in relevant animal models
