Precision BioSciences doses first patient in DMD gene trial

A hospital in Arkansas just treated the first patient in a bold new gene-editing attempt. The target is the gene behind Duchenne muscular dystrophy. The disease weakens boys' muscles from early childhood and significantly shortens their lives. On Aug. 24, doctors at Arkansas Children's Hospital gave the first dose of a gene-editing therapy called PBGENE-DMD. It's made by Precision BioSciences, and designed to fix the underlying mutation, not just slow the disease down.

Current DMD treatments mostly manage symptoms. Steroids preserve muscle function a little longer, and physical therapy helps too. A couple of newer gene therapies add a partial, working copy of the missing protein. PBGENE-DMD works differently. It uses a gene-editing tool to cut the mutation out of a patient's own DNA. The goal is a permanent fix, not an ongoing treatment. That's a much bigger swing, and a riskier one. Editing DNA inside a living person doesn't have a long safety record yet.

Keep reading — what to watch

Will this first patient tolerate the treatment safely? That's really the only question this stage of the trial can answer. Phase 1/2 trials like this one exist to catch dangerous side effects before anyone asks whether a therapy actually works. Real evidence on whether PBGENE-DMD actually helps is still years away. This is only the first of many patients the company needs to dose.