Aggregated News
On May 26, Tracy Atteberry checked in to the hospital at the National Institutes of Health in Bethesda, Md. The 57-year-old has an ultrarare inherited disease that hobbles his immune system so that the most innocuous of germs could kill him. Now he was going to receive a potential cure in the form of a gene editing treatment that had never before been tried in patients.
But, in a tale that gene therapy experts say is too common, Mr. Atteberry and one other patient, a Canadian teenager, may be the only ones ever to get this treatment. As has happened over and over when gene therapies are developed, the company that made it, Prime Medicine, is no longer offering it.
The problem is economics. The science is there to cure Mr. Atteberry’s condition, a subtype of chronic granulomatous disease, or CGD. But it costs so much to develop a treatment and get it approved that even when a company decides to start along this path, it often abandons the effort.
In part, that is because there are so few patients...