FDA Draft Guidance: Leveraging Prior Knowledge to Accelerate Cell & Gene Therapies (Genome Editing)

FDA's Center for Biologics Evaluation and Research (CBER) released draft guidance titled 'Leveraging Prior Knowledge in Development of Human Gene Therapy Products Incorporating Genome Editing.' When finalised, the guidance will allow sponsors to use publicly available information and established platform knowledge — including CMC data, nonclinical study results, and clinical information — to streamline regulatory submissions. Key scope: human gene therapy products that use genome editing (e.g., CRISPR) in human somatic cells. The guidance complements: (1) FDA's Plausible Mechanism Framework for ultra-rare disease therapies; (2) the April 2026 draft guidance on genome editing safety standards and NGS-based off-target assessment. Together, these form a three-part gene therapy regulatory framework. Sponsors must still provide a scientific rationale demonstrating applicability of prior knowledge. Public comments are open — stakeholder input is actively encouraged before finalisation.