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RSNN SIG Expert Meeting Report - In vivo gene editing
The RSNN Special Interest Group Advanced Therapies Expert Meeting on in vivo gene editing took place on Tuesday 15 July 2025. A group of 30 expert from both private and public organisations, including researchers, regulators, representatives from pharmaceutical companies, patient representatives, policy advisors, and consultants came together to discuss the rapidly evolving field of in vivo editing.
Discussions focused on three main themes: off-target effects, non-clinical model systems, and quality criteria for manufacturing. Participants agreed that there is currently no harmonized regulatory framework for off-target assessment, highlighting the need for standardized, transparent methods and patient participation in risk evaluation.
Key recommendations of the meeting included the development of harmonized criteria for off-target effect detection, creation of a European registry for long-term monitoring of off-target effects, and adoption of parallel learning strategies where non-clinical and early clinical data are integrated. Early scientific advice and practical case-based regulatory guidance were identified as crucial for aligning expectations between developers and regulators. The meeting underscored the importance of transparency, collaboration, and patient involvement in shaping future regulatory pathways.
The full meeting report is available for download here.