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The final guidance provides recommendations for developing gene therapy products incorporating genome editing of human somatic cells.
FDA published a final guidance document, Human Gene Therapy Products Incorporating Human Genome Editing, on Jan. 30, 2024. The document provides recommendations for developing gene therapy products incorporating genome editing (GE) of human somatic cells. Recommendations include information needed in an investigational new drug application for the assessment of the safety and quality of GE products. This information pertains to product design, manufacturing and testing, nonclinical safety assessment, and clinical trial design.
According to the agency, the use of human GE has increased, which has resulted in rapid development of gene therapy products that incorporate GE. FDA advises that the risks of these products is not well understood. The guidance document offers ways to assess the safety of these products. The agency states in the document that it may reevaluate its recommendations as the field evolves.
The guidance document defines human GE as, “a process by which DNA sequences are added, deleted, altered, or replaced at specified location(s) in the genome of human somatic cells, ex vivo or in vivo, using nuclease-dependent or nuclease-independent GE technologies. Human gene therapy products incorporating GE are referred to as human GE products throughout this guidance. FDA evaluates human GE products using a science-based approach weighing the benefits and risks of each product. The benefit-risk profile for each product depends on the proposed indication and patient population, the extent and duration of therapeutic benefit achieved, and the availability of alternative therapeutic options. Some of the specific risks associated with GE approaches include off-target editing, unintended consequences of on-target editing, and the unknown long-term effects of on- and off-target editing.”
Considerations addressed in the document include GE methods, type and degree of genomic modification, and GE component delivery method. Chemistry, manufacturing, and controls recommendations are also discussed.