*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

  • Lawrence Tam Group Leader Vector Engineering Group, Gyroscope Therapeutics

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

  • Pooja Agarwal Executive Director Gene Therapy Research, BioMarin

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

  • Niren Murthy Professor of Bioengineering, University of California at Berkeley

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

  • Leah Byrne Assistant Professor of Ophthalmology, The University of Pittsburgh

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

6:30 pm End of Conference Day One