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October 15, 2025

Scientists are inventing treatments for devastating diseases. There’s just one problem.

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.

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/