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

  • Leszek Lisowski PhD MBA, Unit Head Translational Vectorology Research Unit (TVRU), Children’s Medical Research Institute, The University of Sydney

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

  • Dave Chu Director, Capsida Biotherapeutics

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

  • Eric Kelsic Founder & Chief Executive Officer, Dyno Therapeutics

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?

  • Ben Deverman Director - Vector Engineering, The Broad Institute of MIT & Harvard

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 Optimizing rAAV Vectors Through Design And Characterization

  • John Thompson Vice President, Genomics and Computational Biology, Homology Medicines Inc.

Synopsis

• All components of vector payload affect functional impact

• Assays of vector behavior must be carried out in as realistic a model system as possible

• Assays must be carefully designed and calibrated to avoid signals from artifacts

12:00 pm 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

12:30 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

1:00 pm Lunch & Networking

NEW KIDS ON THE BLOCK: NOVEL VIRAL VECTOR SYSTEMS ADDRESSING OLD PROBLEMS

2:00 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.

2:30 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

3:00 pm Non-Replicative HSV Vectors: Overcoming the Gene Delivery Challenge for Local Chronic Diseases

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

3:30 pm Afternoon Refreshments & Networking

NECK TO NECK: NON-VIRAL VECTORS THAT ARE CHALLENGING VIRAL VECTORS

4:00 pm 3DNA Vector Platform: Overcoming Obstacles and Looking Ahead

  • Bob Getts Chief Platform Technologies Officer, Code Biotherapeutics

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

4:30 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

5:00 pm Chairs Closing Remarks