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Just Swap It Out

Newer approaches may boost the number of conditions that gene therapies can treat, the Associated Press reports. It adds that there are currently about half a dozen approved gene therapies in the US and that there are some 400 gene therapy trials underway.

The main challenge facing gene therapy has been delivering the needed version of the gene to patients' cells, the AP adds, noting that therapies for blindness deliver the treatment right to the eye, while other therapies isolate patients' blood cells, alter them in the lab, and then return them to patients with a fixed gene.

Now, the AP says, other viral vectors, including HIV and AAV, are being explored as delivery vehicles. With this type of approach, it says that companies could largely just swap out the genes targeted to treat different diseases. "The only thing that's changing is the payload, the gene," Chris Mason, Avrobio's chief scientific officer, tells the AP.

The AP notes, though, that challenges remain. "Every time we think we have overcome the safety issues, we continue to butt up against them," Peter Marks from the US Food and Drug Administration tells it. "I have confidence that we will work through them ... but we can't get too sure of ourselves."