Agenda
9:20 am Chair’s Opening Remarks
UNDERSTANDING THE REAL RISK VERSUS PERCEIVED RISK OF AAV GENE THERAPY TREATMENTS
9:30 am Examining a Variety of Biomarkers as Reliable Indicators of Liver Toxicity
Synopsis
• Understand the mechanism and relationship between transaminase increases liver damage more clearly
• Explore additional indictors of liver toxicity and evaluating their efficacy to accurately measure liver toxicity due to AAV vectors
10:00 am Mastermind Session: Enhancing Patient Safety through Effective Communication and Optimized Regulatory Protocols: Bridging the Gap between Drug Developers, Clinicians, and Regulatory Bodies
Synopsis
• Improving communication between drug developers, clinicians and regulatory bodies to improve patient safety
• Evaluating the potential toxicity concerns for individual clinical trials and properly educating clinical trial teams
10:30 am Morning Refreshments & Speed Networking
Synopsis
Our structured networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in gene therapy and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against the industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond.
11:30 am Panel Discussion: Discovering the Risk: Benefit Ratio of AAV Gene Therapies & Life Limiting Indications
Synopsis
• Understanding the difference in adverse event tolerance between the different target populations
• Highlighting the balance between the risk associated with AAV gene therapy and the severity and life threatening capacity of the patient's disease
• Curating individual risk: benefit ratios for individual patients to understand the need for the AAV gene therapy treatment
EXPLORING HOW ANIMAL MODELS CAN BE USED TO OPTIMIZE AAV SAEFTY
12:00 pm Extrapolating AAV Data from Experimental Animals to Aid in Designing Human Studies
Synopsis
• Examining the impact of animal model efficacy and toxicity data on translating AAV therapy to the clinic
• Utilizing data from mice and nonhuman primates in predicting human responses to AAV therapy
• Strategies to optimize insights from experimental animals to understanding AAV safety in humans
12:30 pm Lunch & Networking
1:30 pm Panel Discussion: Understanding the Pitfalls Associated with Monkey Models
Synopsis
• Assessing the mechanism for how monkeys manifest toxicity in response to AAV vectors
• Elucidating the mechanism for how liver damage occurs in monkeys
• Exploring the differences between the monkey and human cellular immune response to better inform AAV safety understanding
HIGHLIGHTING THE IMMUNOGENICITY CONCERNS LINKED TO AAV VECTORS
2:00 pm Towards a Safer and More Durable Gene Therapy Vector for Pompe Disease
Synopsis
- Strategically designed gene therapy vector to target tissues that are difficult to treat by ERT and cross-correcting gene therapies
- Identification of transcriptional enhancers for increased expression in key muscle tissues
- Engineered GAA protein with improved activity, stability, cellular uptake and immunogenic profile
2:30 pm Strategizing Methods to Mitigate Against Neutralizing Antibodies in AAV Gene Therapy
Synopsis
• Leveraging bacterial enzymes to circumvent neutralizing antibodies
• Exploring novel approaches to dosing patients with pre-existing immunity
• Optimizing neutralizing antibody assays to improve standardization
3:00 pm Afternoon Refreshments & Networking
4:00 pm Complement-Mediated Thrombotic Microangiopathy Associated with Systemic AAV Gene Therapy
Synopsis
• Review of the complement system and pathophysiology of thrombotic microangiopathy (TMA)
• Learnings from the TMA experience in AAV gene therapy programs to date
• Mechanistic hypotheses regarding TMA risk
• Practical considerations around TMA surveillance, prophylaxis and management
4:30 pm Exploring Methods of Immune Mitigation and Models for Early Prediction
Synopsis
• Delving into the various strategies to understand and mitigate the immune response
• Identifying models to ensure early prediction is achieved
• Comparing old models to novel models to envision the future of mitigation and prediction
HIGHLIGHTING NOVEL/ALTERNATIVE MODELS TO DEMONSTRATE AAV SAFETY
5:00 pm Mastermind Session: Identifying Alternative Models to Reduce the Need for Animal Models & Exploring the Challenges Associated with Providing Accurate Safety Signal Information
Synopsis
This mastermind will give each group the same set of questions surrounding this idea, and then answers will be collated at the end of the session and distributed to all attendees post-conference to gain insights into current perspectives in the field, and suggestions and ideas of relevant actions we can take as a collective to navigate misconceptions in gene therapy
• Exploring the viability of organoid chip technology to help aid in AAV safety signals
• Delving into the challenges of utilizing 3D organ models to better define the AAV safety profile in the liver
• Discovering other in vitro models available and assessing how well they can inform AAV safety in comparison to animal models
5:30 pm Chair’s Closing Remarks
