Morning Briefing
Summaries of health policy coverage from major news organizations
First Attempt To Use Gene Editing To Cure Patients With Rare Disease Offers Sobering Reality Check
Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, although it's too soon to know if this will help. Preliminary results suggest that two men with a rare disorder now have a corrective gene at very low levels, which may not be enough to make the therapy a success. (2/7)
The first attempt at using genome editing to treat and cure patients with a rare, inherited disease has produced disappointing results in a small clinical trial. Despite the setback, Sangamo Therapeutics (SGMO), the biotech developing the genome-editing therapy, intends to keep trying. Transient improvements observed in a single patient suggest a more potent version of its treatment — already being readied for use — might be more effective, the company said. (Feuerstein, 2/7)
