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Gene-editing technology clinical progress and risks

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2026-09-18 14:07 UTC → 2026-09-30 15:04 UTC · added ~~removed~~

Advancements in gene-editing technologies, including CRISPR-Cas9, base editing, and prime editing, are demonstrating significant therapeutic potential. Research from the Broad Institute indicates these tools are being utilized in over 25 clinical trials to target conditions such as leukemia, high cholesterol, and rare genetic diseases. Despite these clinical successes, research published in Nature by Columbia University highlights substantial risks regarding the use of these technologies in human embryos. While next-generation base editing has shown the ability to accurately modify DNA in single-cell embryos, the process also produces unpredictable changes. These biological risks currently prevent the clinical application of embryo editing. Recent developments have introduced a new technique termed ‘DNA pencil’ to address these safety concerns. Unlike traditional CRISPR-Cas9, which acts as molecular ‘scissors’ by cutting both strands of the DNA helix and risking genetic loss or mosaicism, this approach utilizes base editing. By attaching an enzyme to a modified Cas9 protein, researchers can convert a single chemical letter of the genome without breaking the double helix. In testing, this method was used to deactivate the NANOG gene and target the PCSK9 gene associated with heart disease. While these applications produced significantly fewer unintended genomic alterations than conventional methods, the technology still faces limitations, as some tested embryos exhibited chromosomal anomalies or unintended edits. In the commercial sector, CRISPR Therapeutics is working to evolve its capabilities beyond its approved medicine, Casgevy, by developing in-vivo editing processes. Additionally, its CTX460 program has entered phase 1 trials for alpha-1 antitrypsin deficiency. Adding to the debate on embryo modification, Origin Genomics CEO Cathy Tie has advocated for the development of technology to repair defective genes in human embryos. Using stem cells from donated embryos with life-threatening mutations, the startup aims to identify tools to prevent the inheritance of certain diseases.

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