Ornit Chiba-Falek

Company: Duke University/CLAIRIgene

Job title: Professor & Chief, Division of Translational Brain Sciences/Co-Founder

Seminars:

3:00PM Targeting Organ & Cell Type Specific Responses to Enhance the Efficacy & Safety of Gene Therapy 3:00 pm

• Comparison of CNS, systemic, and tissue-specific immune responses to AAV delivery • Challenges in achieving effective CNS penetration while managing immune activity • Case studies on optimizing dose strategies based on target organ immunogenicityRead more

day: Conference Day 2

12:30PM Fireside Chat: Exploring Complement Activation – Strategies for Safe & Precise Gene Therapy 12:30 pm

• Discussing the risk of complement activation and avoiding immune-related adverse events • Understanding how we can avoid off-target transgene expression in unintended tissues and cell types and fine-tuning of gene expression • Open Audience Discussion – identifying current knowledge gaps and collaborative opportunities to advance understanding of complement activationRead more

day: Conference Day 1

9:00AM Keynote Panel: Gene Therapy Immunogenicity: Challenges, Innovations & the Road to Safe & Effective Therapies 9:00 am

• Overview of recent advancements in gene therapy, focusing on immunogenicity as a critical bottleneck in development and clinical success • Novel tools for immunogenicity assessment, including predictive modeling, biomarkers, and innovative vector designs to minimize immune responses • Collaborative strategies to overcome immunogenicity challenges, streamline regulatory approvals, and deliver safe and effective therapies to…Read more

day: Conference Day 1