7:30 am Coffee & Registration
8:20 am Chairs Opening Remarks
NAVIGATING CHALLENGES IN EARLY VECTOR SELECTION AND DESIGN
8:30 am The Art of Generating AAV Capsid Libraries: A Critical Step Towards Bioengineering the Next Generation of Clinical Vectors
Synopsis
• The various types of capsid libraries, including methods of making them
• Size vs fitness – the critical balance between library size vs quality of individual variants within the library
• Weighing the pros and cons of different types of libraries against each other
• New machine learning in silico methods of library design
9:00 am Practical Considerations in Early Design to Improve Your Vector’s Profile
Synopsis
• Combined library and rational design strategies achieve improved tissue tropism and potency
• Small changes in vector design can cause large differences in stability and potency
• Lessons and challenges of engineered vectors on process development, manufacturability, and vector quality
9:30 am Machine-Guided Design Of Best-In-Class AAV Capsids For Eye And CNS Delivery
Synopsis
• Accelerating AAV engineering through in vivo NHP screening and machine learning
• Methods for measuring transduction to determine which capsids are best in class
• Properties of capsids optimized for CNS and ocular delivery
10:00 am How Can We Develop Vectors That Enable Human Gene Therapy Using Models We Cannot Trust?
Synopsis
• Capsid engineering is a proven approach for generating AAV vectors with enhanced capabilities
• In vivo selections often lead to species-specific enhancements resulting in questions about translation
• Cross-model enhancements and mechanism of action studies provide increased confidence about the translation potential of engineered vectors
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 industry leaders and establish
meaningful business relationships to pursue for the rest of the conference and beyond.
11:00 am Morning Refreshments & Networking
11:30 am Incorporating Manufacturability as an Early Checkpoint for Design and Selection of Scalable Vector Designs
Synopsis
• Why scalability and manufacturability represent some of the largest issues faced in the development of Gene Therapies
• How incorporating manufacturability in vector designs can enable seamless upscaling to large scale production
• How vector design can enable increased productivity of the production process with regard to yields (titer) and quality (percentage full capsids)
• Increasing collaboration between early design and CMC teams to smoothen downstream transitions
12:00 pm Challenges and Opportunities in Gene Therapy Engineering
Synopsis
• Payload engineering to optimize specificity, immunogenicity, potency and regulatability
• Capsid selection, rational design and high-throughput capsid engineering
• Potential approaches to enable dosing immunocompetent patients
• Novel Therapeutic modalities and complementary approaches to AAV gene delivery
12:30 pm Lunch & Networking
NEW KIDS ON THE BLOCK: NOVEL VIRAL VECTOR SYSTEMS ADDRESSING OLD PROBLEMS
1:30 pm Chemogenetics: The Chimeric Ion Channel Technology Pushing the Boundaries of Gene Therapy
Synopsis
• Brief overview of the current options to regulate your transgene product
• Controlling the activity of the transgene product using ion channels
• Addressing the current challenges and looking at the way forward.
2:00 pm SVec: Key To The Success of the Next Generation Gene and Immunotherapies Targeting Genetic Disorders and Autoimmune Diseases
Synopsis
• Amarna’s simian virus 40 (SV40)-based SVec vectors are safe by design and non-immunogenic & tolerogenic in humans, inducing long-term transgene expression and opening the possibility of repeated administration with SVec vectors to deliver the same or different transgenes to target tissues
• The non-immunogenicity in humans and capacity to induce immune tolerance to transgene proteins render SVec highly attractive for use in gene replacement and immunotherapies
• SVec is an attractive alternative to AAV vectors for use in clinical in vivo gene therapy
2:30 pm Harnessing the power of HSV-1 gene delivery platform to address local chronic neuronal disorders
Synopsis
• Overview of using HSVs as gene therapy vectors
• Showcase of the HSV platform
• Common problems faced by HSV systems and the path to overcoming them
3:00 pm Poster Session & Networking
NECK TO NECK: NON-VIRAL VECTORS THAT ARE CHALLENGING VIRAL VECTORS
3:30 pm 3DNA Vector Platform: Overcoming Obstacles and Looking Ahead
Synopsis
• Overview of 3DNA vector platform for gene delivery
• Common problems faced when delivering genetic medicines and the path to overcoming them
• Advantages of 3DNA over conventional gene delivery methods and other nonviral approaches
4:00 pm Looking at How RBC Extracellular Vesicle Derived Vectors are Solving Challenges in the Non-Viral Space
Synopsis
• The fundamentals of engineering RBC derived non-viral vectors
• How RBC derived vectors can solve challenges faced by other vectors
• Addressing current challenges and the way forward