Conference Day Two - January 25, 2023

8:55 am Chair’s Opening Remarks

8:59 am
Innovations in Gene Therapy Manufacturing: Enhancing Efficacy, Purity & Comparability

9:00 am Honing Your Regulatory CMC Dossier Details Upfront & During Screening


  • • Streamlining regulatory submission process by strategically refining CMC dossier contents upfront
  • Elevating safety measures and risk assessments in gene therapy manufacturing including both product and process related impurities
  • Discussing practical approaches to seamlessly integrate comparability studies into the CMC dossier

9:30 am Employing State-of-the-Art Analytical Techniques to Demonstrate Analytical Comparability in a Global Setting


  • Designing a risk-based analytical comparability strategy for gene therapy programs
  • Understanding components of an analytical comparability exercise and criteria for demonstrating comparability
  • Supporting the interplay between established and routine assays and novel, characterization assays when conducting analytical comparability exercises

10:00 am Exploring Different Approaches & Expectations in Proving Comparability in a Global Setting


  • Clarifying the FDA Expectations on Repeat Studies for Non-Clinical Studies Between and Before Phase 3
  • Exploring Different Global Approaches to Comparability between the FDA, EMA and other Regulatory Bodies
  • Understanding the Depth and Timelines Needed for Comparability Data as Gene Therapy Programs progress to phase 1 and 2 When Resource Burden Increases

10:30 am Morning Break & Networking

11:00 am Shrinking the Rate-Limiting Step: How Can We Streamline Potency Assay Parameters for Trial Efficiency while Maintaining Proof of Concept


  • Understanding how and when to use potency assays within regulatory requirements to prove your product isn’t variable
  • Evaluating the need for potency assays with associated quantification vs. simply proving gene function and delivery as a strategy to reduce the burden of a matrix of multiple assays
  • Exploring current stances on flexibility towards demonstrating comparability with potency assays and potential next steps for simplifying this process

11:30 am Panel Discussion: What Are the Challenges & Future Directions for Potency Assay Development?


  • Defining the requirements of a matrix of potency assays from early to late stage regulatory
  • Delving into strategies for aligning assay development with evolving regulatory expectations to ensure compliance and product quality
  • Gaining insights into the future directions of potency assay development to cutting-edge technologies and methodologies and how these new approaches might alter regulatory routes

12:00 pm Refining Process Control Strategy & PPQ for Successful Pivotal-Led Pre-BLA & Pre-MAA Submissions

  • Deirdre Harwood Sr. Director, Regulatory Affairs CMC, Ultragenyx Pharmaceutical Inc


  • Development of an aligned pre-PPQ position in Phase 3
  • Explore real-world case studies of pivotal-led BLA submissions and MAA engagement with FDA and EMA
  • Highlight best practices leading to regulatory success and expedited approval pathways for gene therapy products

12:30 pm Regulatory Strategies & Platform Approaches to Accelerate Development of Gene Therapy

  • Catherine Campbell Vice President - Regulatory Affairs, Quality & Good Practice Compliance Ccampbell@ 4dmt. Com, 4D Molecular Therapeutics


  • Leveraging a platform approach to streamline gene therapy development
  • Discussing ideal vector choice, transgene payloads, promoters, and strategies to leverage platform vectors to treat multiple disease indications.
  • Pre-IND, nonclinical, CMC, clinical and regulatory strategies and approaches to accelerate development

1:00 pm Lunch & Networking

2:00 pm Roundtable Discussion: Exploring CMC Regulatory Challenges from Product Testing, Vector Characterization & Beyond


  • Exploring the route to standardized testing for more consistent vector characterization
  • Integrating evolving technology into CMC regulation
  • Ensuring more robust vector characterization sequencing technologies to ensure capsid stability for viral vectors and potency assay conditions are met under the new potency assay guidance
  • Setting acceptance criteria for characterization testing depending on target product profile

2:30 pm Exploring New Methods to Assess Genetic Stability of Cells Beyond Karyotyping & FISH Analyses

  • Saumyaa Saumyaa Senior Scientist & Associate Director - Research, Cell & Gene Editing Therapy Safety, AstraZeneca


  • Exploring New Methods to Assess Genetic Stability of Cells Beyond Karyotyping & FISH Analyses
  • Understanding more precise methods used to assess genetic stability than karyotyping when the mutation is not established
  • Developing novel genomic stability and functional assays
  • Establishing a gold standard for assessing genetic stability to ensure accurate comparison

3:00 pm Chair’s Closing Remarks & End of Conference