State of the Ecosystem

Individualized genetic therapies have treated dozens of patients in a small number of countries, and their regulation forced into existing regulatory paradigms. Regulatory authorities have acknowledged that existing models may not be sufficient, as evidenced by the recent introduction of US FDA Guidance for a Plausible Mechanism Approval Pathway. However, there is little practical experience in these new frameworks.

Our Focus

The N1C Regulatory Workgroup aims to synthesize others’ experiences in submitting and ultimately gaining approval for individualized genetic therapies. This includes collating regulatory feedback into helpful hints, troubleshooting issues across the community, and facilitating future submissions.