7:30 am Coffee & Registration

8:20 am Chairs Opening Remarks


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


• 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


• 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


• 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


• 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


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 Leveraging Scale to Screen for Novel Capsids & Promoters


  • A cross-species approach to identify novel capsids and further layering of modifications in specific capsid regions.
  • A circular RNA-based promoter screening platform for gene therapy.

12:00 pm Incorporating Manufacturability as an Early Checkpoint for Design and Selection of Scalable Vector Designs


• 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 Advances in Manufacturing Best-in-Class rAAV Vectors


• Towards more cost-effective AAV gene therapies: modulating transcription of rep and cap in packaging plasmids to increase the ratio of full capsids

Towards safer AAV gene therapies: identifying and characterizing impurities derived from upstream materials

12:40 pm Challenges and Opportunities in Gene Therapy Engineering

  • Daniel Cohen Vector Optimization Research Leader, Spark Therapeutics


• Payload optimization to improve potency, specificity, and reduce immunogenicity

• High-throughput capsid screening for novel or expanded tropism

• Strategies to overcome neutralizing antibodies

• Complementary approaches to AAV delivery

1:10 pm Lunch Refreshment Break & Hosted Lunch with Mirus Bio


2:10 pm Chemogenetics: The Chimeric Ion Channel Technology Pushing the Boundaries of Gene Therapy


• 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.

2:40 pm SVec: Key To The Success of the Next Generation Gene and Immunotherapies Targeting Genetic Disorders and Autoimmune Diseases

  • Peter de Haan Chief Scientific Officer & Co-Founder, Amarna Therapeutics


• 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

3:10 pm Harnessing the power of HSV-1 gene delivery platform to address local chronic neuronal disorders


• 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

3:40 pm Poster Session & Networking


4:10 pm 3DNA Vector Platform: Overcoming Obstacles and Looking Ahead


• 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

4:40 pm Looking at How RBC Extracellular Vesicle Derived Vectors are Solving Challenges in the Non-Viral Space


• 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

5:10 pm Chairs Closing Remarks