8:50 am Chair’s Opening Remarks
EXPLORING SHORT TERM IMMUNOGENICITY AND THE IMPACT ON DURABILITY
9:00 am Examining the Impact of the Capsid on Short Term Durability
Synopsis
• Comparing the impact of different capsids on short term immune reaction based on clinical data
• Exploring strategies to mitigate the initial reaction
• Discovering how capsid engineering can be used to minimize the immune response
9:30 am Determining how Immunosuppressants can be Optimized to Achieve Gene Therapy Durability
Synopsis
• Mitigate initial reaction to transgene to achieve short term durability
• Does it enable patients with prior AAV immunological responses to be doses with gene therapy treatments?
• Is controlling T regulatory cells, and purely T cell depletion the main thing to impact the antibody response?
10:00 am Discovering the Advantages of Using Novel Immunosuppressants to Boost Durability
Synopsis
• Leveraging novel immunosuppressants to promote re-administration and improve transgene expression
• Exploring how the timing of immunosuppression treatment affects the response to the transgene
• In depth characterization of T cell responses to the transgene
10:30 am 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.
11:00 am Morning Refreshements
11:30 am Durability and Immunogenicity of Inhaled SP-101, A Novel AAV2.5T Gene Therapy for Cystic Fibrosis
Synopsis
• Understanding the unique durability challenges relating to delivering gene therapies via inhalation to the lung
• Exploring a novel AAV capsid (AAV2.5T) for inhaled delivery of a CFTR minigene (hCFTRdR) for the treatment of Cystic Fibrosis
• Assessing data to understand the durability of hCFTRdR transgene expression and associated immunogenicity
ESTABLISHING A WELL-DEFINED DEFINITION OF DURABILITY AND THE ASSOCIATED REGULATORY PROTOCOL
12:00 pm Exploring the Strategies Available for Measuring Durability
Synopsis
• Assessing biomarkers as an accurate method of measuring durability
• Establishing clearly defined clinical end points
• Reviewing the ease and accuracy of using biopsies to measure protein expression
12:30 pm Mastermind Session: Understanding ‘’What is Durability?’’ and Finding the Middle Ground for Demonstrating Durability to Regulatory Bodies
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
• Determining a definition for durability
• Discovering FDA/EMA insights
• Navigating what clinically measurable indictors the FDA want to see
• Understanding how to gather durability data without a trial needing to last 10 years
• Support for following up with patients’ long term
1:00 pm Lunch & Networking
TRANSLATIONAL SUCCESS FROM MODELS TO HUMAN TRIALS
2:00 pm Discovering the Optimal Strategies to Achieve Durability in Preclinical Studies to Best Navigate the Transition into Clinical Trials
Synopsis
• Learning how to interpret preclinical data to improve translational success
• Understanding how to better predict the effects seen in humans
• Exploring how novel technologies can be combined with animal models to improve durability prediction
2:30 pm Exploring a Horse Model Trial and the Translatability into Humans for Osteoarthritis (OA)
Synopsis
• Critical target for OA gene therapy
• Pre-clinical data in a horse model of OA
• Phase 1 clinical trial and 1-year analyses in OA subjects
3:00 pm Discovering how Best to Predict the Translational Success of AAV Gene Therapies
Synopsis
• Analyzing pre-clinical and clinical data to assess the accuracy of durability predications of AAV gene therapies • Learning about novel methods to enhance durability prediction to minimize delays in clinical trials
• Comparing different pre-clinical models to identify the optimal model for durability
3:30 pm Afternoon Refreshments & Networking
4:00 pm Evaluating the Durability of AAV8 Gene Therapy in Mouse and NHP
Synopsis
• Long term expression in mouse
• Mixed durability results in NHP
• Evaluating different factors that may impact durability