*All times are in EDT
HIGHLIGHTING THE NEW FRONTIER OF VECTOR ENGINEERING
8:20 am Chairs Opening Remarks
8:30 am Strategies Towards Optimising AAV Gene Therapy Approaches For Targeting the Retina
Synopsis
- Landscape of investigative gene therapies for retinal diseases
- Challenges associated with AAV vector delivery to target the retina and the long-term effectiveness of therapies
- Current strategies to optimize AAV delivery and transgene expression in the posterior retina
9:00 am Developing Next Generation Gene Therapy Vectors for the Treatment Of Rare Genetic Diseases
Synopsis
- Capsid, transgene, and process optimization for AAV gene therapy candidates
- Assessing process and product quality impact on potency, kinetics, and durability
- Selecting appropriate ex vivo and in vivo models to predict clinical safety and translatability
9:30 am Re-engineering the Building Blocks of Gene Therapy: Improving Efficacy, Safety & Manufacturability of AAV With the ART Platform
Synopsis
- Novel capsids with improved tissue targeting & immunogenic profile
- Novel promoters modulate transgene expression in specific cell types
10:00 am Developing Next Generation Gene Therapy Vectors
Synopsis
- Improving promoter tissue specificity and towards inducibility and tunability
- Next-gen capsids for vascularised tissues and beyond
- Systemic re-administration on the horizon?
10:30 am Morning Break & Structured Networking
VIRAL VECTOR TRACK
INCREASING TISSUE & CELL TYPE VECTOR TROPISM
12:00 pm AAV-ligand Conjugates, a New Capsid Platform For Improved Gene Therapy Delivery
Synopsis
- AAV capsids from 1st and last generations limitations (efficiency and safety) are related to the nature of the capsid composition
- Chemical conjugation of AAV capsid alter dramatically the biology of AAV leading to improve bio distribution, transduction and immunological profile
- Chemical conjugation of AAV is a versatile and scalable new capsid technology
12:30 pm In Vivo Gene Editing With Generide Technologies
Synopsis
- Novel GeneRide technique for enhanced transgene editing in vivo;
- Novel AAV capsids for liver disease indications
- Novel AAV capsids with enhanced CNS
NON-VIRAL VECTOR TRACK
ADVANCES IN DNA DELIVERY PLATFORMS FOR NON-VIRAL GENE THERAPY
12:00 pm Harnessing the Power of a DNA-Delivery Platform to Address Chronic Inflammatory Diseases
Synopsis
- Advantages of DNA-Delivery, including safety, potential flexibility and manufacturing efficiency
- Demonstration of functionality with XT-150, addressing critical areas where other delivery modalities of IL-10 therapy have been ineffective
- Clinical program results for XT-150, a novel immunomodulatory gene therapy for osteoarthritis pain in Phase 2B development
12:30 pm Solving Key Delivery Challenges involved in Multiple Genetic Medicines
Synopsis
- Overview of synthetic non-viral 3DNA® platform and how it’s used
- How 3DNA can be used as a non-viral delivery for gene therapy
- Advantages of using Code Bio’s 3DNA approach for gene therapy
1:00 pm Lunch & Networking Break
THE PURSUIT OF VECTORS TO REDUCE & EVADE IMMUNE RESPONSE
2:30 pm Overcome the Immunogenicity Limitations To Reach The Largest Population
Synopsis
- Current limitations of gene transfer with AAV vectors
- Next generation vectors to overcome pre-existing humoral immunity to increase the reach of AAV gene therapy
- Next generation vectors and new methods to overcome post-treatment humoral immunity toward repeated vector administration
3:00 pm Overcoming Immunogenicity Challenges of AAV Gene Therapy Vectors
Synopsis
- AAV gene therapy elicits a complex immune response, involving innate and adaptive B and T cell responses that can affect safety, efficacy and the ability to re-dose AAV vectors
- Pre-existing anti-AAV neutralizing antibodies preclude many patients from being able to receive AAV gene therapy
- I will present unpublished data from studies in mice, nonhuman primates and humans using our ImmTOR tolerogenic nanoparticle to mitigate de novo AAV immunogenicity as
well as data on a novel and differentiated IgG protease to address pre-existing antibodies
OPTIMIZING NANO-PARTICLE APPROACHES IN THE NON-VIRAL FIELD
2:30 pm Analyzing Thousands of Nanoparticles in Vivo Using High-throughput Screening
Synopsis
- High-throughput screening
- Identification of non-liver delivery vehicles
- Discussion of next steps for the field of delivery
3:00 pm Non-viral Delivery of Gene Editing Enzymes With New Polymers and Lipid Nanoparticles
Synopsis
- Gene editing with the RNP
- New strategies for endosomal disruption
- New types of lipids for making lipid nanoparticle based mRNA delivery vectors
3:30 pm Afternoon Break & Networking
OPTIMIZING NEXT GENERATION VECTOR DESIGN
4:00 pm Panel Discussion: Assessing Capsid Engineering Techniques – Searching for the ‘Perfect’ Capsid
Synopsis
- Discussing different techniques to engineer capsids
- How can we assess which capsids are not only effective but also scalable in manufacturing?
- Will there ever be a ‘one size fits all’ capsid?
- Highlighting progress in preclinical and clinical trials with optimized AAV capsids
5:00 pm Machine-Guided Design of Optimized AAV Vectors
Synopsis
- AAV capsid engineering can be greatly accelerated using AI methods
- Capsids are optimized for specific indications and administration routes
- Progress towards higher efficiency, specificity, manufacturability and improved safety
5:30 pm Presentation Slot Reserved for Event Partner
6:00 pm scAAVengr: A Transcriptome-based Pipeline for AAV Engineering With Single-cell Resolution
Synopsis
- We have developed scAAVengr, a single-cell AAV engineering pipeline to simultaneously quantify and rank efficiency of competing AAV vectors across all cell types in parallel, in the same primates
- Transcriptome based-quantification of AAV-mediated gene expression allows for precise evaluation of AAV performance and determination of vector tropism, as well as the development of novel AAV vectors for rapid and efficient clinical translation