This past spring, a biotech company announced the first use of a new gene-editing technology in people to fix an errant gene that causes a severe immune disorder. In June, a baby born with a life-threatening metabolic disorder was allowed to leave the hospital after a six-month sprint by scientists to create a bespoke treatment for him. And increasingly, a generation of “bubble babies” born without immune defenses are nearing their teenage years after receiving a one-time experimental gene therapy in early childhood.
Scientists are inventing treatments for devastating diseases. There’s just one problem.
Therapies that target genetic illnesses at their root are no longer on the horizon. They are here. More are coming. But even as a growing suite of gene therapy tools are changing individual patients’ lives, many are getting stuck in a medical purgatory becausethey don’t fit the model for turning breakthroughs into accessible treatments.
https://www.washingtonpost.com/health/2026/01/11/gene-therapy-rare-diseases/
