The FDA has approved rebisufligene etisparvovec-hopf, a one-time gene therapy for eligible children with Sanfilippo syndrome type A, marking the first approved treatment for this rare inherited neurodegenerative disease. The evidence suggests better preservation of cognitive development than seen in an untreated natural-history group, but the pivotal comparison was not a conventional randomized placebo-controlled trial.
A first-in-disease Phase 1 trial found deep B-cell depletion and encouraging remission signals after treatment with the experimental bispecific antibody A-319 in people with active lupus. The approach could eventually offer a simpler alternative to individualized CAR-T therapy, but the study involved only 12 patients and cannot yet establish effectiveness.
An experimental inhaled RNA medicine successfully reduced a selected inflammatory protein inside the lungs in an early human trial, suggesting that gene-silencing medicines may eventually be delivered directly to lung tissue.
A genetically modified pig kidney worked for 271 days in a man with kidney failure, allowing him to avoid dialysis before he later received a human donor kidney.
