As CRISPR-based therapies become ever complex, drug developers urgently need to innovate the analytical tools available to effectively characterize their safety and efficacy profiles and agree upon gold standards moving forward. We are also witnessing increased regulatory scrutiny, with the FDA closely studying off-target analysis conducted ahead of the approval of Casgevy, prior to finalizing guidance on genome editing therapies in January.
To keep up with these growing analytical demands, the 2nd CRISPR-Based Therapy Analytical Development Summit is returning to unite 50+ key figures across Analytical Development, CMC & QC domains, all with the common goal of developing more precise, accurate, and sensitive analytical assays.
Expert discussions will highlight analytical innovation with both traditional and disruptive CRISPR-based therapies, and span in vivo and ex vivo delivery, covering the following core themes:
- Characterization of guides, nucleases, and full drug constructs
- Robust non-clinical and CMC assay development, including biodistribution studies, potency assays, and on-/off- target sequencing detection, nomination, and prediction
- Meeting regulatory expectations for analytical data to successfully support IND submissions
If you are working to develop, characterize, and validate more robust analytical tools to enable the progression of your early R&D work into a clinical therapy, this is your collaborative forum to equip you with the latest innovation, case studies, and actionable insights to level up your analytical efforts.
