Biotech company Prime Medicine received a regulator’s green light to start testing its next-generation gene editing candidate in humans. On 29 April, the US Food and Drug Administration approved the biotech’s Investigational New Drug application, which gives Prime the go-ahead to test its first product candidate, PM359, in a phase 1/2 clinical trial in pediatric and adult patients with chronic granulomatosis disease (CGD).
CGD is a rare immunodeficiency caused by mutations in genes that affect phagocyte function by compromising nicotinamide adenine dinucleotide phosphate (NADPH) production and the cells’ ability to produce pathogen-killing reactive oxygen species. This renders patients susceptible to severe life-threatening bacterial and fungal infections. Hematopoietic stem cell transplants are the only definitive cure for CGD.
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FDA clears prime editors for testing in humans.
Nat Biotechnol 42, 691 (2024). https://doi.org/10.1038/s41587-024-02264-6
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Published: 17 May 2024
Issue Date: May 2024
DOI: https://doi.org/10.1038/s41587-024-02264-6
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