A new technology for “editing” defective genes has raised hopes for a future generation of medicines treating intractable diseases like cancer, cystic fibrosis and sickle-cell anemia.

Such drugs could home in on a specific gene causing a disease, then snip it out and, if necessary, replace it with a healthy segment of DNA.

Drugs of this type wouldn’t hit the mass market for years, if ever; pharmaceutical firms are only now exploring how to make drugs using the gene-editing technology, called Crispr-Cas9. But the approach offers tremendous potential for developing new treatments for diseases caused by a mutated gene.

“What if you could go right to the root cause of that disease and repair the broken gene? That’s what people are excited about,” says Katrine Bosley, chief executive of privately held Editas Medicine. Its projects include developing a gene-editing drug treating one type of Leber congenital amaurosis, a rare disease that causes blindness in children.

Research and controversy

Crispr-Cas9 isn’t the only technology capable of editing genes, but researchers consider it easier to use than other methods, says Dana Carroll, a professor of biochemistry at the University of Utah School of Medicine, who helped pioneer another gene-editing approach called zinc finger nucleases.