SeQure DX™ Risk Assessment Services Align With FDA Guidance For Gene-Edited Human Gene Therapy Products

The US FDA released new guidance in 2024 for human gene therapy products that incorporate human genome editing, outlining critical considerations for product development, non-clinical and clinical studies, and the information required for Investigational New Drug (IND) filings. When designing gene editing components, developers should optimize them to reduce off-target genome modification, and a description of this process and its rationale should be included in the IND. Understanding population genomic heterogeneity, highly sensitive off-target detection, and the use of multiple orthogonal assessment methods are emphasized. It is also important to evaluate human cell types from multiple donors and describe off-target biologic impacts.

Gain insights into the FDA's expectations for gene-edited human gene therapy products by downloading this scientific brief.