Conference Day One
Wednesday, June 4
8:00AM Registration & Coffee
8:20AM Chair’s Opening Remarks
9:00AM Keynote Panel: Gene Therapy Immunogenicity: Challenges, Innovations & the Road to Safe & Effective Therapies
Synopsis
• 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 patients worldwide
STRATEGIC INSIGHTS INTO REPEAT DOSING & IMMUNE MANAGEMENT FOR GENE THERAPIES
9:30AM Strategic Approaches to Implement Repeat Dosing
Synopsis
• Develop standardized, sensitive assays to interpret TAb and NAb responses across AAV serotypes, leveraging lessons from companion diagnostics for consistent therapeutic class interpretation
• Identify clinically impactful anti-AAV titers, assess the relevance of systemic titers for local therapies, and explore dosing strategies over low-titer responses as seen with Hemegenix
• Establish bioanalytical approaches to mitigate immune responses and enable repeat AAV dosing, ensuring diagnostics align across different products in the same class
10:00AM Case Study: Next-Generation AAVs for CNS Targeting: Enhancing Precision While Reducing Liver Tropism & Immunogenicity
Synopsis
• Exploring novel capsid modifications that improve blood-brain barrier crossing and increase therapeutic efficacy in neurological disorders
• Strategies to substantially detarget the liver, lowering systemic toxicity and reducing immune activation
• How advanced AAV engineering can mitigate immune responses, improve patient tolerability, and enhance long-term therapeutic success
10:30AM Morning Refreshments & Speed Networking
OVERCOMING IMMUNE HURDLES BY CLEARING ANTIBODIES, OPTIMIZING CAPSIDS & ADVANCING SAFETY TO UNLOCK THE FULL POTENTIAL OF GENE THERAPIES
11:30AM Case Study: Exploring Novel Solutions to Clear Pre-Existing Antibodies & Expand Patient Eligibility for Treatment
Synopsis
• Exploring innovative techniques to address pre-existing antibodies that can block treatment
• Practical approaches to antibody removal and immune modulation for enhanced therapeutic outcomes
• Advancing treatment options for patients otherwise ineligible due to immune barriers
12:00PM Fulfilling the Promise of Next Generation Capsids to Optimize Efficacy, Safety & Scalability for Real World Therapeutic Applications
Synopsis
• Exploring novel capsids designed to improve therapeutic delivery and reduce immune responses
• Evaluating their potential to address challenges in muscle-targeted gene therapies
• Can these innovations achieve targeted delivery with lower or higher doses while mitigating risks?
12:30PM Fireside Chat: Exploring Complement Activation – Strategies for Safe & Precise Gene Therapy
Synopsis
• 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 activation
1:00PM Lunch & Networking
ENHANCING TRANSLATIONAL SUCCESS BY TACKLING IMMUNE VARIABILITY, DIAGNOSTICS & INNOVATIVE MANAGEMENT IN GENE THERAPY
2:00PM Addressing Variability in Immune Responses Across Species to Improve Translational Accuracy
Synopsis
• What are the limitations of current animal models in predicting human immune responses?
• How can we improve our understanding of interspecies variability, and what innovative methods are being explored to enhance translatability?
• Share examples of successful or unsuccessful translations of preclinical findings to the clinic
2:30PM Pharmacodynamic & Clinical Activity of 4D-310 in Fabry Patients with Low Capsid-Neutralizing Factors Detected Using a Cell Based Assay
Synopsis
• Examining the pharmacodynamics of 4D-310 and its ability to overcome immune responses in Fabry patients with low capsid-neutralizing factors
• Understanding the clinical activity of 4D-310 and its potential to improve patient outcomes in the context of Fabry disease
• Discussing the role of cell-based assays in detecting capsid-neutralizing factors and optimizing gene therapy strategies for patients with pre-existing immunity
3:00PM Afternoon Refreshments & Poster Session
4:00PM Round Table Discussion: Targeted Immune Management Strategies to Mitigate Antibody Responses & Improve Gene Therapy Precision
Synopsis
• Exploring the use of enzymes to reduce antibodies before drug administration
• Balancing immunogenicity control with therapeutic efficacy to optimize gene therapy results
• Investigating how viral vector design impacts both safety and therapeutic effectiveness
4:30PM Leveraging Immunogenicity Insights & Companion Diagnostics to Predict Risks & Enhance Gene Therapy Safety
Synopsis
• Understanding how transgene immunogenicity impacts patient safety and therapy outcomes
• Leveraging advanced diagnostics to predict and mitigate safety risks
• Reviewing current assays, modalities, and tools designed to address safety concerns in the patient