A 57-year-old man with a rare inherited disease was the only patient to receive a groundbreaking gene editing treatment. Now, the company behind it has abandoned efforts due to high costs, leaving him and another patient in limbo.
A gene therapy for a rare inherited disease was tested on two patients at the National Institutes of Health in May 2023. The 57-year-old man, Tracy Atteberry, had a condition that makes him susceptible to deadly infections. The treatment, developed by Prime Medicine, showed promise but ultimately failed due to economic constraints.
The company behind the gene therapy is no longer offering it, citing high production costs as the reason for its abandonment. This is not an isolated incident; experts say that this tale of gene therapy development and abandonment is too common. Despite the potential cure, Atteberry and another patient remain in a state of uncertainty, awaiting news on the future of their treatment.
Source: https://www.nytimes.com/2025/09/22/health/gene-editing-rare-diseases-fda.html