Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing
The FDA issued a draft guidance for industry regarding the safety assessment of genome editing in human gene therapy products through the use of next-generation sequencing (NGS).
Manufacturers, sponsors, and developers of human gene therapy products utilizing genome editing technologies, as well as laboratory functions performing NGS analysis.
The issuance of this draft guidance suggests an increasing regulatory focus on standardized methodologies for identifying off-target genomic modifications. For developers, this likely indicates that NGS will be a prioritized tool for safety characterization. Regulatory teams may need to prepare for more specific documentation requirements regarding sequencing depth, bioinformatic pipelines, and the sensitivity of assays used to detect unintended genetic alterations.
Regulatory and bioinformatics teams should evaluate current NGS protocols against these draft safety assessment benchmarks. Quality units should review data integrity and validation standards for genomic sequencing used to identify off-target effects. Clinical operations may need to incorporate these assessment parameters into trial safety monitoring plans.
This is the draft guidance for industry, Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing
Open in openFDA / FDA.gov