8:20 am Chair’s Opening Remarks
LEARNING FROM CLINICAL EXPERIENCES TO ESTABLISH LONG TERM DURABILITY
8:30 am Assessing the durability of a long-term clinical study for Neovascular AMD
Synopsis
- Delving into the strategies to minimize inflammation while having durable responses
- Reflecting on 3 years of clinical data and biomarker data to provide evidence for durability
9:00 am Learning from Gene Therapy Treatments in Immunoprivileged Places to Apply Systemically
Synopsis
• Understanding how the immune response is control when minimal
• Reviewing clinical findings in eye/ear gene therapy trials
• Identifying elements that can be applied to systemic gene therapies
9:30 am Key Learnings from a Cardiac Targeted Program
Synopsis
• Exploring the evidence for limited turnover of cardiomyocytes in children and adults
• Reviewing 2-3 year gene expression follow-up in Phase 1 Danon disease AAV9 trial
• Navigating a long term follow up of adult patients with Danon Disease
10:00 am Strategies to Improve the Durability of Gene Therapies for Hemophilia A
Synopsis
• Assessing clinical data to understand the durability of gene therapies for Hemophilia A
• Comparing different delivery methods and the impact on durability
• Understanding the optimal strategy to achieve maximum durability
10:30 am Morning Break & Networking
INVESTIGATING HOW TO OVERCOME LONG TERM IMMUNOGENICITY TO IMPROVE DURABILITY
11:00 am Navigating Long Term Immunological Issues Relating to the Transgene
Synopsis
• Understanding the long term humoral and cellular responses to the transgene over time
• Comparison of delivering the transgene into an immunologically privileged place or systemically
• Clinical reports of transgene immune response impact on durability
• Understanding the immunologic response of different transgenes and the resulting impact
11:30 am Exploring the Multiple Impacts of MTOR on Gene Therapy Durability
Synopsis
• Assessing the impact of MTOR to facilitate redosing
• Discovering how MTOR can lead to sustained levels of the transgene
• Leveraging MTOR to overcome liver inflammation in response to AAV
12:00 pm Lunch & Networking
1:00 pm Exploring Novel Vectors/Technologies that show Promise for Redosing to Reduce the Impact of Durability
Synopsis
• Assessing data from novel vectors to understand if redosing is a viable option
• Discovering novel technologies to increase the possibility of redosing and the impact on durability
• Navigating long term challenges related to redosing
1:30 pm Exploring Immune Responses that Eliminate Expression at a Later Stage
Synopsis
• Understanding what triggers late-stage immune responses
• Discovering methods to identify late-stage triggers to aid in evading the immune response to maintain durability
• Novel strategies to tackle spontaneous immune responses
2:00 pm Exploring the Non-Immunological Considerations Relating to Durability
Synopsis
• Understanding the importance of the target cell population
• Delving into the impact of cellular stress on durability
• Highlighting the potential of genome integration and the impact on durability
• Navigating patient lifestyles to boost durability
2:30 pm Afternoon Refreshments & Networking
LEVERAGING ADVANCEMENTS IN VECTOR OPTIMIZATION TO MAXIMIZE DURABILITY
3:30 pm Comparing Ubiquitous and Specific Promoters and the Effect on Durability
Synopsis
• Understanding the additional safety concerns associated with ubiquitous promoters
• Exploring how to best manipulate promoters to achieve lasting durability
• Navigate strategies to better predict promoter selection on durability
4:00 pm Panel Discussion: Exploring Vectors Beyond AAV and the Unique Durability Challenges and Opportunities They Present
Synopsis
• Assessing immunogenicity concerns relating to non-viral vectors
• Understanding their potential to enable redosing and minimize durability concerns using lentivirus vectors
• Explore clinical updates of non-viral vectors and lentivirus vectors to improve durability