7:45 am Registration and Morning Networking

8:05 am Chairperson’s Opening Remarks


8:15 am Considerations Towards Development of In Vivo Gene Therapies – a Risk Based Approach


  • In vivo Gene Therapy: key principles and major cmc challenges
  • Understanding the linkage between CMC and clinical outcomes

8:45 am Scaling the Manufacturing Process: The Importance of Small Scale Models in Supporting Comparability Assessments


  • Animal models provide a critical framework for the preclinical evaluation of gene therapy products.
  • The vectors generated for these nonclinical studies are typically made at a lab scale using a process that differs from the eventual clinical and commercial manufacturing processes.
  • Often, these lab processes utilize techniques that are not scalable, resulting in impurity profiles that can drive differences in critical attributes and clinical performance of products – including in immune response, dosing, and toxicity. Ideally, manufacturing processes should be scaled with representative material, to support accurate product analysis and characterization through each phase of development.
  • This presentation will discuss methods for comparability assessments to support translation and scaling of production processes.

9:15 am Determining if Improved Impurification Techniques Can Impact Potency

  • Kyle Grant Director of Vector Production, Voyager Therapeutics


  • Assess the impact of commercial purification techniques on a drug substance purity and potency in vitro and in vivo.
  • Evaluate the impact and influence of viral delivery on clinical safety.
  • Assess the current purification techniques and the advantages of novel methodologies.

9:45 am Making (anti) Sense out of AAV vectors


    • Competing platforms for AAV manufacturing
    • Plasmid-derived AAV vectors have co-packaged DNA contaminants
    • DNA contaminants lead to backbone-derived antisense RNAs
    • dbDNA as an alternative starting material
    • dbDNA eliminates plasmid backbone sequences, leads to improved vector purity and potency

10:15 am Morning Coffee Break & Networking

11:15 am Virtual Presentation: Considerations when Adapting Methodology During Process Development

  • Van Hoang Head of Analytical & Quality Control, Spark Therapeutics


  • Factors to consider for comparability assessments when transitioning to different methods.
  • Discuss the critical need for re-testing and the available strategies to bridge methods when samples are limited.
  • Consider the risk mitigation tactics when methods evolve during process development.

11:45 am Bottlenecks in Comparability Testing: Round Table Discussions

Join a smaller group of delegates to discuss some of the common bottlenecks to comparability design and execution and strategize solutions to navigating these critical challenges

Novel Methods Being Adopted For Empty Vs Full Characterization

Michael Rosconi, Associate Director, Regeneron

  • Discussion of new, not-so-new, andre-purposed methods to assess empty/full capsid ratio.
  • When and where to apply said methods during AAV product development.
  • How many tools do you need in the belt? Importance of orthogonality when characterizing empty and full capsids

Strategies to Support Material Retention and Crisis Management

Susan Sleep, Director Analytical
Development, AGTC

  • Early processes often have significant material constraints, especially for inprocess samples.
  • Comparability studies can require large volumes to demonstrate statistical improvement.
  • We will identify practical ways to optimize the use of these precious materials and strategize to get the most important data to report.

Challenges and Constraints in Gene Therapy CMC

Markus Haindl, Head Gene Therapy
Technical Development, Roche

  • Navigate the challenges of limited productivities in Gene Therapy development.
  • Better understand the linkage between CMC and clinical outcomes.
  • Discuss the strategies to effective and sustainable scale up of gene therapy development.

12:45 pm Lunch & Networking


2:15 pm Virtual Presentation: Adapting State-of-the-Art Analytical Techniques to Demonstrate Analytical Comparability in AAV Gene Therapy Programs


  • Outline common challenges to AAV analytical comparability packages.
  • Identify strategies to execute analytical comparability to account for challenges.
  • Introduce new technologies, including MAM, that can be leveraged to bolster comparability packages.
  • Discuss risk mitigation when selecting methodology to prevent material waste and preserve sample retains.

2:45 pm Case study: Lessons Learned When Bridging Historic Data for Comparability Assessments


  • Evolution of analytics from early to late phase.
  • Examples of best practices in bridging data and solutions.

3:15 pm Virtual Presentation: Creating in-house Reference Standards To Support In Industry Wide Comparisons And Benchmarking Methods

  • Dawn Henke Senior Scientific Program Manager, Standards Coordinating Body


  • Discover the different approaches being used to build assay standards.
  • Understand the progress being made to support method validation and comparisons.
  • Discuss the reference materials currently being used to develop and qualify more relevant in house reference standards.

3:45 pm Afternoon Break & Networking


4:15 pm Tailoring Gene Editing Therapies from Discovery through Preclinical Development Using Analytical Methods For Benchmarking Small CRISPR Cas Nucleases

  • Greg Hoffman Vice President Platform & Discovery, Arbor Biotechnology


  • Arbor is developing genetic medicines using a wholly owned portfolio of proprietary gene editing enzymes, including CRISPR cas nucleases with small sizes and unique properties that enable breakthrough applications.
  • Arbor nucleases are mined from metagenomes, engineered using a high-throughput pipeline, and optimized for activity in therapeutically relevant cellular contexts.
  • We will discuss analytical approaches for comparing nuclease activity spanning search, bacterial screening, mammalian cell screening, and preclinical, development.

4:45 pm Early Stage Qualification Of Assays: Extending the Development Of iCIEF-Western Method From Mab to AAV to Support Extended Characterization

  • Kun Lu Senior Staff Scientist, Regeneron


  • How to select assays that will be futureproof and support robust comparability analysis.
  • How to define release specification criteria that will support reproducibility of results late stage.
  • Discuss the analytical methods to support extended characterization:
    • CE-Western (Size-based) and iCIEF-Western (charge-based) are both developed in Regeneron to support mAb size and charge variants in comparability studies.
    • AAV anti-individual VP protein antibodies were developed to apply iCIEF-Western to AAV extended characterization.
    • Specific anti-individual VP protein antibodies detection provides identification and quantitation to assess each individual VP protein charge heterogeneity.

5:15 pm Chairperson’s Closing Remarks