Pre-Conference Focus Day

Tuesday, March 25

8:30 am Registration & Light Breakfast

9:00 am Opening Remarks & Group Introductions

  • Morten Sogaard President, Research & TechOps, Astellas Innovation Management

9:30 am Effective Strategies for Transitioning from Rare to Common Diseases to Expand Gene Therapy Horizons

  • Morten Sogaard President, Research & TechOps, Astellas Innovation Management

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

  • Van Hoang SVP, Head of Analytical & Quality Control, Spark Therapeutics

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

  • Craig Malzahn Senior Vice President, Technical Operations, REGENXBIO

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

  • Kathleen Kirby Chief Operating Officer, Merlin Therapeutics
  • Van Hoang SVP, Head of Analytical & Quality Control, Spark Therapeutics
  • Morten Sogaard President, Research & TechOps, Astellas Innovation Management
  • Craig Malzahn Senior Vice President, Technical Operations, REGENXBIO
  • Marriane Lopez Senior Research Director, Duke University

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

2:30 pm End of Pre-Conference Focus Day