FDA Describes Its Approach to Genome-Edited Products

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In a blog post, Robert Califf and Rita Nalubola discuss the agency’s approach to the use of genome-edited products.

In a FDAVoice blog post dated Jan. 18, 2017, FDA Commissioner Robert M. Califf, MD, and Senior Policy Advisor Rita Nalubola detailed the agency’s science-based approach to genome-edited products, stating that while the agency is excited about the potential of these products, it is focused on ensuring the safety of regulated products that use genome-editing technologies.

Genome-editing technologies, which can be used to introduce, remove, or substitute one or more specific nucleotides in an organism’s genome, have been considered for use in the treatment of diseases such as HIV, cancer, and rare diseases. According to Califf and Nalubola, genome-edited products may be used in a variety of medical, food, and environmental sectors but pose potential risks that include affects to individual genomes and potential environmental and ecosystem impacts.

The agency, therefore, is working to ensure the use of these technologies remains safe. Multiple government agencies, including the White House Office of Science and Technology Policy, FDA, the US Environmental Protection Agency, and the US Department of Agriculture’s Animal and Plant Health Inspection Service, are involved in the effort to ensure the effectiveness of the regulatory system for biotechnology products. The agencies have established a national strategy and published a framework for the regulation of biotechnology products. 

The agency is also working with international regulators. FDA’s Center for Biologics Evaluation and Research (CBER) is a member of the International Pharmaceutical Regulators’ Forum. Members identify and exchange information on issues of mutual concern and undertake targeted regulatory cooperation activities.


Regarding their specific use in biologics, CBER has policies in place to evaluate gene therapy products. Zinc finger nuclease (ZFN)-mediated gene editing is being applied in clinical trials in the United States. National Institutes of Health’s (NIH) Recombinant DNA Advisory Committee (RAC) has discussed the first clinical protocol to use CRISPR/Cas9-mediated gene editing. “The potential for 'off-target' effects such as insertions or deletions at unintended genetic loci has been identified by experts in the field as a key concern,” stated Califf and Nalubola.

The agency remains diligent, according to Califf and Nalubola. “The expansive scope of intentional genomic alterations using modern genome editing technologies has triggered debate on fundamental ethical and social issues, which will continue to influence public opinion and acceptance of genome editing applications. Even as FDA implements necessary steps for effective regulation to ensure the safety of products, the role of broader, inclusive public discussion involving multiple constituencies (e.g., scientists, developers, bioethicists, and public interest and community groups) to address the larger societal considerations should not be overlooked.”

Source: FDA