Comment from Anusha Sriraman
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Summary: A group of scientists and researchers from various biotechnology and technology development companies (including Pacific Biosciences, Praxis Molecular Solutions, Catalent, Sangamo Therapeutics, and Form Bio) expressed broad support for the FDA's draft guidance on NGS-based genome editing safety assessments. They recommend specific technical enhancements, such as establishing a limit of detection (LoD) for assays, requiring explicit bioinformatics version control, and encouraging validation against reference datasets.
We are a group of scientists and researchers from industry, and technology development organizations with expertise in adeno-associated virus (AAV) biology, next-generation sequencing (NGS), and gene therapy product characterization. We appreciate the opportunity to comment on the FDA’s draft guidance document entitled “Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing” (April 2026) (hereafter, “the Guidance”).
We broadly support the Guidance and welcome the FDA’s recognition of NGS as a critical tool for assessing genome editing safety in human gene therapy products. The Guidance arrives at a timely moment: the field is rapidly advancing, and clear regulatory expectations for NGS-based on- and off-target edit site assessment are both needed and appropriate. We are particularly encouraged that the Guidance avoids being overly prescriptive with respect to specific assay platforms or sequencing technologies, instead directing sponsors to justify the adequacy and sensitivity of their chosen approach. This flexibility is appropriate given the pace of innovation in sequencing and bioinformatics.
Our specific comments follow the structure of the Guidance document.