Comment from Testavec Ltd
AnonymousSupportAcademic
Summary: The commenters, representing TestAVec Ltd and Brunel University of London, support the FDA's draft guidance but argue for a more comprehensive "three-pillar" framework. They recommend integrating vector integrity, genome integrity, and biological consequence into the risk assessment to better account for vector heterogeneity and the functional effects of genomic alterations.
We submit comments on Draft Guidance "Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing" (Docket No. FDA-2026-D-1255)
on behalf of Testavec Ltd and Brunel University.
Our comments offer 5 recommendations:
1. Recognize comprehensive vector characterization as an important complement to genome editing analysis.
2. Encourage the use of long-read sequencing to evaluate vector heterogeneity, rearranged genomes and packaged non-vector nucleic acids where scientifically appropriate.
3. Acknowledge that orthogonal molecular methods, including RNA sequencing, targeted PCR and functional genomic analyses, may be required to interpret complex genomic alterations.
4. Recognize the emerging value of validated human cell-based functional assays capable of assessing the biological consequences of genomic alterations beyond their structural identification.
5. Encourage integrated evaluation of vector integrity, genome integrity and biological consequence as complementary components of risk assessment.
The full comment letter, including references, is attached.