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# Human Genome Editing Product Development Guide
- URL: https://www.fdaweb.com/human-genome-editing-product-development-guide/
- Published: 2024-01-29T12:00:00.000Z
- Updated: 2026-09-14T14:21:26.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156232

FDA has issued a [guidance](https://www.fda.gov/media/156894/download?ref=fdaweb.com) entitled “Human Gene Therapy Products Incorporating Human Genome Editing.” The document provides recommendations about the information that should be included in an IND in order to assess the safety and quality of the investigational genome editing (GE) product, such as product design, product manufacturing and testing, nonclinical safety assessment, and clinical trial design. The agency says human GE is 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.”

FDA notes that the interest in GE as a scientific technology for treating human diseases has increased substantially, and there has been rapid development of gene therapy products relying on GE. “While the potential of such products for the treatment of human disease is clear, the potential risks are not as well understood,” the guidance says. “To assist in the translation of these products from the bench to clinical trials, this guidance includes recommendations for how to assess the safety and quality of these products and address the potential risks of these products.”

Additionally, the guidance says that 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.” Because human GE is a rapidly evolving field, FDA says it may revise its recommendations on developing GE products as they gain information on the safety of such products.