Conference Day Two
Thursday, June 5
8:00AM Registration & Coffee
8:45AM Chair’s Opening Remarks
OVERCOMING PRE-EXISTING IMMUNITY TO AAV: STRATEGIES & INNOVATIONS FOR TREATING AAVPOSITIVE PATIENTS IN GENE THERAPY
9:00AM Case Study: Effective Strategies to Identify, Reduce, & Manage Pre- Existing Antibodies to Expand Patient Eligibility in Gene Therapy
Synopsis
• How prevalent is pre-existing AAV immunity in different patient populations?
• Discuss screening and stratification techniques to identify AAV-positive patients
• Explore approaches like plasmapheresis, vector engineering, and alternative serotypes to bypass antibody response
9:30AM Exploring Novel Approaches to Mitigate Gene Therapy Immunogenicity
Synopsis
• What are the latest advances in immunosuppression protocols to mitigate immune responses to the AAV vector and the transgene product?
• Discuss the risks and benefits of these strategies, including patient safety considerations and immune system recovery
• Sharing examples demonstrating success or challenges with immunosuppressive treatments
10:00AM VTX-PID Mediated Optimal Depletion of Anti-AAV Neutralizing Antibodies in Human Subjects Allows Expanded Patient Eligibility for Future AAV-Based Gene Therapies
Synopsis
• How to optimize patient benefit and optimal strategy for neutralizing antibody-positive subjects
• Sharing clinical data demonstrating success and challenges in AAV Nabs positive subjects with PK/PD modeling
• Exploring the potential of these therapies to expand patient eligibility and potentially improve patient outcomes
• Implementation in real life and providing access for all AAVs and patients considering benefit-risk
10:30AM Morning Refreshments & Networking
MAXIMIZING TRIAL SUCCESS BY STANDARDIZING ASSAYS & FOSTERING COLLABORATIVE PATHWAYS IN GENE THERAPY
11:30AM Maximizing Clinical Trial Success with the Advantages of Generic Assays for Consistency & Accuracy
Synopsis
• Discuss the potential benefits of adopting standardized assays across trials, such as improved data comparability, streamlined regulatory review, and reduced development timelines
• Share examples where generic assays have facilitated advancements in therapy evaluation or approval
• Address challenges in ensuring that standardized assays remain relevant across different therapeutic areas
12:00PM Fireside Chat: Driving Standardization through Collaborative Pathways to Improve Gene Therapy Outcomes
Synopsis
• What are the current gaps or barriers to assay standardization in gene therapy?
• How can collaboration between industry, academia, and regulatory bodies accelerate the adoption of standardized assays?
• Brainstorm ways to ensure standardized assays remain adaptable to evolving therapeutic innovations
12:30PM Roundtable Discussion: Optimizing Patient Recruitment with Advanced Screening Methods to Ensure Trial Success
Synopsis
• Analyze the effectiveness of current screening methods, including genetic testing and immune response assessments
• What are the potential benefits of adopting more comprehensive or less invasive screening approaches?
• Share examples of innovative screening practices that have improved recruitment outcomes
1:00PM Lunch & Networking
COMPARING DIFFERING ADMINISTRATION ROUTES FOR GENE THERAPIES
2:00PM Fireside Chat: Optimizing Gene Therapy Delivery through Different Administration Routes
Synopsis
• Compare and contrast delivery methods like intraocular, sub-retinal, and emerging techniques
• Discuss challenges unique to each route, including surgical complexity, patient tolerance, and immune considerations
• Brainstorm ideas for minimizing invasiveness while maintaining delivery accuracy
2:30PM Comparing Sub-Retinal vs. Intra-Vitreal Delivery Methods to Maximize Safety & Precision in Gene Therapy
Synopsis
• Analyze the pros and cons of sub-retinal delivery (high precision but invasive) and intravitreal injections (less invasive but higher inflammation risk)
• Share real-world examples or studies highlighting outcomes with each approach
• Explore technical advancements and innovations aimed at improving these delivery routes
3:00PM Targeting Organ & Cell Type Specific Responses to Enhance the Efficacy & Safety of Gene Therapy
Synopsis
• 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 immunogenicity