The FDA has approved Kresladi, the first gene therapy for severe leukocyte adhesion deficiency type I (LAD-I). By adding a working ITGB2 gene to a child's own stem cells, it gives white blood cells back the grip they need to leave the bloodstream and fight infection, with no donor needed.

The FDA unveils a “plausible mechanism pathway” to speed approval of innovative therapies for ultra-rare diseases, paving the way for personalized and gene-based treatments.

Japanese researchers restored movement in a paralyzed man using reprogrammed stem cells injected into his spinal-cord injury. The treatment, tested in four patients, helped one man stand independently and another regain limb movement, offering early evidence that iPS cell therapies may support neural repair and future paralysis treatments.