Pre-Conference Workshop Day: Tuesday, September 17
7:30 AM Check In & Coffee
Workshop A
8:30 am A 101 in Potency Assays
Synopsis
- Establishing potency assays relevant for your therapeutic activity
- Developing potency measurements that reflect the product’s mechanism of action
- How early should potency assay development begin?
- How should you adapt your matrix approach through early- to late-stage development?
- Recommendations for assay design and validation, including the importance of using reference materials and standards
- Dissecting the new FDA guidance on potency assurance: how should these be interpreted and implemented to guide strategy?
- Discussing real-life case studies of potency strategies used to support IND filings
10:30 AM Refreshment Break
Workshop B
11:00 am Developing a Framework for Off-Target Prediction & Detection
Synopsis
- Summarizing the most popular methods for off-target nomination and detection: their relative strengths and weaknesses
- What should I consider when choosing, and how do they complement bioinformatic nomination?
- Using appropriate read depth and window size to generate high assay sensitivities
- Within the current regulatory context, how can these tools be applied for the risk assessment of a gene editing tool?
- Understanding modifications to off-target testing requirements in the recently finalized FDA guidance: how to implement this framework in a real-world situation
1:00 PM Lunch Break & Networking
Workshop C
2:00 pm Examining Analytical Requirements for Lipid Nanoparticle Formulations for CRISPR Delivery
Synopsis
- Highlighting the main formulations of lipid nanoparticles (LNPs) used for CRISPR delivery in mRNA, plasmid, and RNP forms
- How does LNP decoration impact biodistribution and analytical strategy?
- Effectively characterizing cargo, including guide-mRNA ratios and degradation levels
- How to account for inherent variability in LNP variability when designing assays to measure efficacy
- Developing CQAs and phase requirements for product development and manufacture
- Understanding the key regulatory requirements for DS and DP analytical testing, including potency strategy to demonstrate LNP functional activity