Conference Day Two
Thursday, March 27
8:50 am Registration & Coffee
8:50 am Chair’s Opening Remarks
EVALUATING LONG-TERM SAFETY & EFFICACY: SUCCESSES & INSIGHTS FROM APPROVED GENE THERAPIES
9:00 am Panel Discussion: Aligning Development with Patient Access: Integrating Market and Patient Needs Early in Gene Therapy
Synopsis
• Lessons from companies that have effectively aligned their development processes with patient access goals from the outset
• Perspectives from downstream stakeholders on what they need to cover and select gene therapies
• Strategies for incorporating patient needs, pricing, and provider reimbursement considerations into early development stages
9:30 am Session Reserved for: Azzur Group
10:00 am Panel Discussion: Investment Strategies for Non-Viral & Viral Gene Therapy: From Seed Financing to Series C
Synopsis
• Overview of the investment requirements and strategies across different stages of gene therapy development
• Key factors that make gene therapy projects attractive to different types of investors, from venture capitalists to strategic partners
10:30 am Morning Refreshments & Networking
Translation
Clinical
Manufacturing
Optimizing Routes for CNS, Ocular & Inner Ear Disorders to Maximize Efficacy & Minimize Side Effects
Evaluating Long-Term Outcomes & Advancing Gene Therapy with Considerations into Safety & Efficacy
Ensuring Consistency & Reliability in Potency Assays for Gene Therapies - From Development to Standardization
11:30 am Enhancing Delivery of Gene Therapies in the CNS to Improve Patient Outcomes
Synopsis
• Case study examples of extensive studies to determine the optimal route of administration for CNS therapies, comparing intrathecal (IT) and intravenous (IV) delivery methods
• The focus is on maximizing therapeutic delivery to the brain and spinal cord while minimizing systemic exposure, crucial for both gene therapy applications and antisense therapies
12:00 pm Case Study: Advancing AAV Gene Therapy Vector Generation in Retina & CNS using ML-Guided scAAVengr-HUnT Platform
Synopsis
• Leveraging our previous single cell-based scAAVengr pipeline, Avista created a new High, Unbiased Throughput (scAAVengr-HUnT) platform, modified to develop and test highly complex libraries of novel AAV variants
• Avista’s Machine Learning (ML) model can design novel packaging-efficient libraries in unexplored sequence space
• We utilize our ML-Guided scAAVengr-HUnT platform to rapidly engineer efficient AAV vectors with maximized therapeutic value for multiple different tissues, such as retina and CNS
11:30 am Case Study: Improved Evaluation of Long-Term Safety & Efficacy of Lovotibeglogene Autotemcel Gene Therapy
Synopsis
• Implementation of extended follow-up periods and comprehensive monitoring to assess the durability of therapeutic effects and identify late-onset adverse events
• Utilization of novel biomarkers and genomic assays to track gene therapy persistence, expression levels, and potential off-target effects over time
• Incorporating patient-reported outcomes and quality-of-life assessments to evaluate the real-world impact and sustained efficacy of the therapy
12:00 pm Navigating Clinical Development Pathways to Commercial Approval: Pfizer’s Success with BEQVEZ Gene Therapy for Hemophilia B
Synopsis
• Insights into Pfizer’s journey to regulatory approval in the U.S., EU, and Canada, addressing unique challenges and breakthrough solutions for BEQVEZ
• Exploration of risk minimization and safety and efficacy control measures that underpin the commercialization of BEQVEZ
• In-depth examples of product comparability and process characterization that supported BEQVEZ’s development and commercialization as a transformative therapy
11:30 am Manufacturing Case Study: Developing Robust Potency Assays for Gene Therapy Products
Synopsis
• Understanding the role of potency assays in regulatory approval and quality assurance
• Strategies for developing sensitive, specific, and reproducible assays
• Addressing variability, matrix effects, and assay validation complexities
12:00 pm In-process, Release & Efficacy Assays: Similarities & Differences
Synopsis
• Should in-process assays mirror release assays?
• Where do matrix effects make the data difficult to interpret?
• If I am in a hurry to prove proof of concept, will that hurt me when it comes time to BLA
Navigating Viral & Non-Viral Systems for Enhanced Precision & Safety
Effective Strategies for Clinical Evidence Generation in Rare Disease Gene Therapies
Managing Risks, Enhancing Collaborations & Overcoming Global Distribution Challenges
1:30 pm Case Study: Identifying the Best Approaches for Viral vs Non- Viral Delivery Methods for Gene Therapies in Rare Diseases
Synopsis
• Evaluating the effectiveness and limitations of viral versus nonviral vectors for delivering gene therapies to targeted diseases
• Tailoring delivery methods to specific disease indications to enhance therapeutic outcomes
2:00 pm Panel Discussion: Addressing Challenges Associated with Viral Delivery Approaches
Synopsis
• Selecting the right delivery system to reduce unintended gene edits and increase accuracy
• Analyzing the benefits and drawbacks of using viral vectors for gene delivery
• Strategies for decreasing immune responses and enhancing the overall safety profile of gene-editing technologies
1:30 pm Clinical Case Study: Executing a Gene Therapy Trial in New Geographies: The Experience with The OAV101 IT Trial In Later Onset Spinal Muscular Atrophy
Synopsis
• Insights into adapting clinical trial protocols and overcoming logistical challenges in diverse regulatory environments for gene therapy trials
• Key takeaways from conducting the OAV101 IT trial for later-onset spinal muscular atrophy in varied international settings
• Strategies to maintain data integrity, patient safety, and regulatory alignment across multiple geographic regions
2:00 pm Panel Discussion: Maximizing Patient Enrollment for Gene Therapy Trials & Strategies for Commercial Success
Synopsis
• Learn effective methods for identifying and enrolling patients in rare disease trials to enhance trial efficiency
• Discover how to balance recruitment strategies with the need to ensure the therapy’s long-term commercial viability
• Explore innovative approaches using technology, partnerships, and advocacy groups to boost patient recruitment and retention rates
1:30 pm Implementing Novel Approaches in Navigating Regulatory, Practical & CMC Considerations
Synopsis
• Best practices for integrating new methodologies into clinical trials and regulatory submissions
• How to work with regulatory agencies to gain approval for novel approaches
• Examples of successful implementation of innovative methods in gene therapy trials
2:00 pm Panel Discussion: Navigating the Complex Supply Chain for Gene Therapies
Synopsis
• Identifying and mitigating risks associated with raw materials and key components
• Collaborating with suppliers, CDMOs, and logistics providers to ensure a reliable flow of materials
• Best practices for maintaining product integrity throughout the supply chain to reduce production delays and maintain product quality
3:00 pm Afternoon Refreshments & Networking
MAXIMIZING INVESTMENT & COMMERCIAL SUCCESS IN GENE THERAPY – STRATEGIC INSIGHTS FOR SECURING FUNDING & ADVANCING DEVELOPMENT
4:00 pm Session Reserved for: VintaBio
4:30 pm Next Steps for Advancing Gene Therapy Commercialization: Takeaways & Action Items
Synopsis
• Key recommendations for companies looking to move forward with gene therapy development and commercialization
• Practical guidelines for navigating regulatory, financial, and market challenges
• Strategies for developing commercially viable therapies that provide long-term value to patients and stakeholders