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
Synopsis
- • 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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?
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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