*All times are in EDT

MANUFACTURING THE NEXT GENERATION GENE THERAPY VECTORS

8:20 am Chairs Opening Remarks

8:30 am Adeno Associated Virus Manufacturing Methods for Improved Efficacy

  • Kyle Grant Director Vector Production, Voyager Therapeutics

Synopsis

  • Assess whether purification techniques of a virus can have a deep impact on the activity or ability to deliver genetic material
  • Evaluate the impact and influence of viral delivery on clinical safety
  • Assess the current purification techniques and the advantages of novel methodologies

9:00 am RNA-seq Guided Multi-gene CRISPR Knockout Further Improves Productivity of High Yielding AAV Producer Cell Lines (PCLs)

Synopsis

  • Evaluation of novel targets focused on viral lifecycle
  • Development of multi-gene KO cell lines
  • Analytical characterization of PCLs and rAAV

9:30 am How to Make Your CMC Regulatory Affairs Colleagues Happy

Synopsis

  • Points for consideration: the CMC dossier section of a clinical trial application or marketing application
  • Common CMC challenges for gene therapy products that may give your CMC regulatory affairs (CMC RA) colleagues a headache
  • What could make your CMC RA colleagues happy?

10:00 am Designing a Platform: Overcoming Challenges in rAAV Manufacturing

  • Jacob Smith Sr Director of Process Development, AskBio

Synopsis

  • Developing a platform process while supporting clinical development & manufacturing
  • Addressing current challenges in manufacturing process development (i.e. material limitations, clinical demand)
  • Cost-effective and scalable process improvements
  • Transition of key regulatory starting materials (plasmid to doggybone DNA)

10:30 am Morning Break & Structured Networking

VIRAL VECTOR TRACK

LEVERAGING NEXT GEN VECTORS FOR A SAFER & MORE EFFICACIOUS PRODUCT

11:30 am AAV Safety Monitoring in Clinical Development

Synopsis

  • Outlining practical aspects of safety monitoring in clinical trials
  • Routine monitoring in follow-ups
  • Processes for mitigation if safety signals are observed

12:00 pm EVADER Immune Suppressive AAV Based Gene Therapy for Repeat Dosing

Synopsis

  • Clinical challenges addressed by EVADERTM technology
  • Scalable production of EVADERTM vectors
  • Anti-Capsid Immune Reduction by EVADERTM vectors

12:30 pm Targeted CODA-receptor Expression For Neurological Disorders Using Next-gen AAV Vectors

Synopsis

  • Brief overview of CODA’s platform
  • AAV capsid selection criteria
  • AAV expression cassette optimization

NON-VIRAL VECTOR TRACK

BETTER UNDERSTANDING EFFICIENCY OF TISSUE TARGETING WITH NON-VIRAL VECTORS

11:30 am GMAB Non-viral, Cell-penetrating Gene Monoclonal Antibody Platform: A Highly Targeted and Unique Approach for Delivery of Therapeutic Nucleic Acids

Synopsis

  • Non-covalent binding and protection of both RNA and DNA payloads
  • Selective targeting of ENT2 transporter and cell penetration into cytoplasm and nucleus
  • Systemic delivery of nucleic acid payloads to ENT2 positive tissues in vivo
  • Optionality for therapeutic nucleic acid targeting in treatment of cancer and musculoskeletal disease

12:00 pm Nonviral and Hybrid Approaches for Gene Therapy

Synopsis

  • Use of the piggyBac transposon system in gene therapy for treatment of severe disease
  • Hybrid approaches for gene therapy in severe urea cycle disorders
  • Non-viral approaches for liver-directed gene therapy and editing

12:30 pm PANEL –Tissue Targeting and De-targetting Approaches With Non-viral Vectors

1:00 pm Lunch Break & Networking

VIRAL VECTOR TRACK

VIRAL VECTOR PLATFORM OPTIMIZATION DEVELOPMENT

2:00 pm Advantages and Disadvantages of Current Lentiviral Vector Platforms

  • Bojiao Yin Associate Director, Process Development, Elevate Bio

Synopsis

  • This talk will focus on current Lentiviral Vector manufacturing platforms for cell and gene therapy applications
  • Elevate bio strategic efforts to develop a GMP-ready, scalable, and robust Lentiviral Vector process based on transient transfection of suspension growing cultures will be presented

2:30 pm AAV9 Engineering Platform & Automation

Synopsis

  • Development of an AAV centered engineering platform
  • Applications of the platform and efforts to automate engineering processes

NON-VIRAL VECTOR TRACK

EXPLORING NOVEL NON-VIRAL PLATFORM DEVELOPMENT

2:00 pm Pioneering Non-viral Gene Therapy Using Red Blood Cell Extracellular Vesicles

Synopsis

  • Introduction to Carmine Therapeutics
  • REGENT platform
  • In vivo data

2:30 pm Non-viral Gene Therapy With C3DNA

Synopsis

  • C3DNA platform
  • Precision engineering
  • Therapeutic applications

3:00 pm Afternoon Break

3:30 pm Long-read Sequencing Fidelity and Genome-wide Integration Assays For rAAV-mediated Homologous Recombination (HR) In Human Hepatocytes

  • John Thompson VP Genomics & Computational Biology, Homology Medicine

Synopsis

  • Identification of artifacts that complicate many AAV integration studies
  • NGS technology solutions to characterize AAV integration
  • Confirmation of specificity of on-target versus off-target integrations

4:00 pm Optimizing AAV Vector Using Data Science

Synopsis

  • Optimization of AAV serotpyes
  • Applying data science research to optimize engineering efforts

4:30 pm End of Conference