Pre-Conference Workshop Day: Tuesday, September 17

Workshop A

8:30 am A 101 in Potency Assays

  • Rachael Cohen Associate Director, Plate Based Assays & Lab Automation, Prime Medicine


  • 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

Workshop B

11:00 am Developing a Framework for Off-Target Prediction & Detection

  • Ankit Gupta Senior Director, Head Of Gene Editing, Affini-T Therapeutics
  • Kai-Yuan Chen Director, Data Science & Tech Platform, Scribe Therapeutics


  • 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

Workshop C

2:00 pm Examining Analytical Requirements for Lipid Nanoparticle Formulations for CRISPR Delivery

  • Carl Co Senior Director - Analytical Sciences, Arbor Biotechnologies


  • 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