Post-Conference Focus Day

Thursday, December 1 2022

NON-AAV VECTORS FOCUS DAY

9:30 am Chair’s Opening Remarks: What Does the Future of Gene Therapy Look Like?

9:45 am Developing a Multiplex Molecular Method for Monitoring DNA Impurities in Lentiviral Vector

Synopsis

  • Discussing the limitations of existing kit-based molecular methods
  • Outlining the development of a quadruplex ddPCR method targeting DNA impurities in lentiviral vector
  • Addressing the current challenges associated with developing a multiplex method

10:15 am Well Characterized Gene Therapies: Comparison of Analytical Strategies for Viral & Non-Viral Vectors

  • Marina Feschenko Senior Director, Analytical Development, Intergalactic Therapeutics

Synopsis

  • Determining the similarities and distinctions in critical quality attributes (CQAs) for viral and non-viral gene therapies
  • Outlining how drugs with different delivery methods and production processes can alter the analytical strategy
  • This presentation will focus on AAV- and C3-DNA-based gene therapies as examples

10:45 am Challenges on the Processing Side for Non-AAV Vectors

Synopsis

  • Stability concerns: discussing issues with the stability of non-AAV vectors and ways to overcome this
  • Safety concerns: non-AAV vectors are often designed to integrate into the genome, which has different safety concerns in comparison to AAV
  • Discussing challenges in purification of non-AAVs, such as preserving infectivity

11:15 am
Networking Break

12:15 pm Panel Discussion: Evaluating the Successes & Failures of Using Non-AAV Vectors & Discussing the Future of Gene Therapy

Synopsis

This panel discussion with the post-conference focus day speakers and chair will give you the chance to speak to peers about the successes and failures of using non-AAV vectors and will be a space to debate the future of gene therapy, and whether non-viral vectors/non-AAVs are going to take over from viral vectors in the future.

12:45 pm Case Study 1: Outlining the Analytical Tools Used to Characterize the Components of Lipid Nanoparticles

1:15 pm
Lunch & Networking

2:15 pm Case Study 2: Reviewing the Lipid Nanoparticle Characterization Toolbox

  • Tom Colace Director, LNP Process Development, Tessera Therapeutics

2:45 pm Case Study 3: Identifying the Best Analytical Approaches to Determine Critical Quality Attributes for Lentiviruses

3:15 pm Case Study 4: Examining New Identity (ID) Assays for Multivalent mRNA Vaccines

  • Jing Zhu Director, Process & Analytical Development, RVAC Medicines

Synopsis

  • Identity (ID) assay required as both in-process and final releasing assays for mRNA vaccines
  • Current progress and challenges of sequence methods as orthogonal approaches for multivalent mRNA ID assay
  • Two newly developed ID methods demonstrated their advantages including shortened lead time, flexibility as a platform method and low requirement for in-house lab capacity

3:45 pm Chair’s Closing Remarks & End of Post Conference Day

Want to View Conference Day One & Two?