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

  • Mark Trusheim Director - Strategic, Tufts Medical Center
  • Francesca Cook Vice President, Global Pricing & Market Access, REGENXBIO
  • Terry Cothran Senior Pharmacy Director, Oklahoma Health Care Authority

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

  • Samir Koirala Head of Genetic, Neurodevelopmental & Epilepsy Disorders, Biogen

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

  • Anjulika Chawla Vice President, Medical Affairs, Clinical Lead, bluebird bio

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

  • Ravi Iyer Chief Scientific Officer, Script Biosciences

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

  • Amanda Placone Senior Director External Innovation and Early Commercial Strategy, Eli Lilly
  • Kathleen Kirby Chief Operating Officer, Merlin Therapeutics

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

  • Kumar Dhanasekharan Senior Vice President & Head of Technical Operations, Voyager Therapeutics

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

  • Jie Li Director, Vertex Pharmaceuticals
  • Kumar Dhanasekharan Senior Vice President & Head of Technical Operations, Voyager Therapeutics

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

5:00 pm Chair’s Closing Remarks

5:15 pm End of 8th Gene Therapy Development Summit