Pre-Conference Focus Day
Tuesday, March 25
8:30 am Registration & Light Breakfast
9:00 am Opening Remarks & Group Introductions
9:30 am Effective Strategies for Transitioning from Rare to Common Diseases to Expand Gene Therapy Horizons
Synopsis
• Analysis of the challenges and opportunities in scaling gene therapy technologies from rare to common diseases, including adjustments in clinical trial design and patient populations
• Examination of strategies to reduce production costs and enhance cost-effectiveness while maintaining high-quality standards for larger patient populations
• Insights into the regulatory hurdles and market strategies required to successfully transition therapies to broader indications and achieve widespread adoption
10:00 am Roundtable: Overcoming the Challenges and & Maximizing Opportunities for Gene Therapy in Common Diseases
Synopsis
• Tackling the technical, regulatory, and financial barriers to gene therapy adoption in widespread conditions
• Strategies to optimize manufacturing processes and reduce costs while maintaining efficacy and quality for larger populations
• Identifying market access opportunities and pathways to ensure successful commercialization and patient access
10:30 am Morning Networking Break
11:00 am CMC Strategies to Scale Gene Therapy for Common Diseases: A Roadmap to Quality and Efficiency
Synopsis
• Process Innovation for High-Volume Production: Leveraging modular production and advanced technologies to meet the demands of large-scale gene therapy applications in common diseases
• Building a Quality-Driven Manufacturing Framework: Establishing robust analytics, in-process controls, and release testing to ensure product consistency at scale
• Integrating Cost-Effective CMC Solutions: Developing strategies for streamlined process validation, risk-based testing, and cost-efficient supply chains tailored for broader patient populations
11:30 am Reducing CoGs & Enhancing Manufacturing Efficiency for Gene Therapies
Synopsis
• Exploration of methods to lower CoGs through process improvements, automation, and scaling up production capabilities while ensuring consistent product quality for common disease
• Examination of quality measures necessary to support manufacturing scalability, including potency assays, purity testing, and regulatory compliance
• Insights from successful case studies and industry best practices in reducing CoGs and enhancing manufacturing efficiency for gene therapies
12:00 pm Lunch & Networking
1:00 pm Foundations for Commercializing Gene Therapies: Key Activities for Successful Transition to Common Disease Markets
Synopsis
• Identify core activities to adapt manufacturing, supply chains, and distribution models for larger patient populations
• Develop effective market access strategies, including pricing, reimbursement, and partnerships, to ensure a smooth entry into common disease markets
• Understand regulatory requirements and navigate challenges for scaling up gene therapy from rare to widespread applications while maintaining product quality and safety
1:30 pm Panel Discussion: Breaking Boundaries: Navigating the Shift from Rare to Common Diseases in Gene Therapy
Synopsis
• Exploring the unique challenges and benefits of expanding gene therapy into more prevalent conditions, including patient access, cost, and treatment scalability
• Evaluating the feasibility of widespread gene therapy applications in common diseases by 2025, along with realistic goals and potential hurdles
• Essential factors for successful translation, including regulatory preparedness, CMC adaptation, and the need for sustainable manufacturing and pricing models to meet higher demand