CONFERENCE DAY ONE | Wednesday, October 22

8:00 am Check-In & Coffee

8:50 am Chair’s Opening Remarks

9:00 am Industry Leader’s Panel: Overcoming Profitability, Analytical Challenges, Emerging Technologies, & Future Directions

  • Scott Jeffers Chief Technology Officer, GenSight Biologics
  • Johnson Varghese Vice President - Analytical Sciences & Development, Beam Therapeutics Inc.
  • Vanessa Strings-Ufombah Executive Director - Assay Development, Chemistry, Manufacturing & Controls, Adverum Biotechnologies

9:45 am Session Reserved for BioRad

10:15 am Presentation & Roundtable Discussion: A Review of Gene Therapy Release Assays & Their Utility

  • Uditha deAlwis Vice President - Analytical Development & Quality Control, Sarepta Therapeutics

10:45 am Morning Break & Speed Networking

Synopsis

As this community re-unites for the 7th time, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections

Bioassays & Molecular Biology

Enhancing Potency Assessment in Gene Therapy for Reliable Assays & Regulatory Success

Overcoming the Challenges of NGS Validation for Gene Therapy Potency & Stability to Improve Consistency

12:15 pm Optimizing Potency Assays for Gene Therapy From Particle Correlation to QC-Friendly Methods

12:45 pm Develop New Assays in Days, Not Months: Get Low-variability Titer, VCN, and Genome Integrity without Tradeoffs

  • Stefanie Kall Ph.D., Senior Staff Product Manager, Countable Labs

12:55 pm Lunch Break & Networking

  • Stefanie Kall Ph.D., Senior Staff Product Manager, Countable Labs

1:55 pm Defining Potency for LNP-Delivered Gene Therapies: From mRNA to Function

Innovative Technologies in Gene Therapy Looking to Advance Vector Characterization & Genome Integrity

2:25 pm Session Reserved for Intertek

2:55 pm Advancing Molecular Techniques for Vector Characterization in Heterogenous Capsid Populations

  • Tyler Lindgren Associate Scientist - Analytical, Research & Developmentand Support, BridgeBio Pharma, Inc.

3:25 pm Afternoon Break & Poster Session

4:25 pm Ensuring Genome Integrity in Gene Therapy with Advanced Tools & Techniques

4:55 pm Ensuring Consistency in Measured DNA Concentration while Transitioning from qPCR to Advanced PCR Methods

Physicochemical Properties

Advancing Analytical Methods for Complex Gene Therapy Products

11:45 am Making AUC GMP-Ready: Optimizing for QC & Regulatory Use

  • Luis Rascon Senior Research Associate, Analytical Development, Astellas Pharma

12:15 pm Aggregation Analysis in AAV: Selecting Fit-forPurpose Assays

  • Margaret Butko Director - Chemistry, Manufacturing & Controls, Assay Development, Adverum

12:45 pm Lunch Break & Networking

1:45 pm Shining Light on Mass Photometry: Applications & Analytical Challenges in Gene Therapy

Defining & Controlling Product Quality to Ensure Safety, Consistency, & Compliance

2:15 pm Session Reserved for Mirus Bio

2:45 pm From Bench to QC: Streamlining Potency Assay Readiness

  • Anmol Kotian Prinicpal Associate Scientist, Solid Biosciences

3:15 pm Afternoon Break & Poster Session

4:15 pm Aging Like Fine Champagne: Ensuring Consistent Quality with Reference Standards

4:45 pm Establishing Infectious Titer Assays with Accuracy & Confidence

  • Win Cheung Senior Director - Analytical Development, REGENXBIO

5:15 pm Chair’s Closing Remarks

5:20 pm End of Conference Day One