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The FDA decides whether a gene-editing medicine can be sold in the United States, and its decisions influence regulators worldwide. In 2026 it made two significant changes: reducing the default number of clinical trials required for approval from two to one, and proposing a framework under which an individualised therapy for an ultra-rare disease might be approved on evidence from very few patients.
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The U.S. Food and Drug Administration regulates gene therapies and gene-editing products through the Center for Biologics Evaluation and Research. It approved the first CRISPR-based medicine in December 2023. In 2026 it reduced the default requirement from two adequate and well-controlled trials to one, and issued draft guidance on individualised therapies for ultra-rare diseases described as a plausible-mechanism framework, under which improvement in a single participant could in defined circumstances support approval.
Sources
- Fierce Biotech · 2026
FDA illuminates new approval pathway for bespoke gene editing therapies ↗