Nature Medicine, Published online: 16 September 2026; doi:10.1038/s41591-026-04636-8

In this single-arm phase 1 trial, an AAV gene therapy carrying the Padua variant of factor IX was well tolerated in 11 adolescents with hemophilia B and led to reductions in annualized bleeding rate.

Cell Death Discovery, Published online: 25 May 2026; doi:10.1038/s41420-026-03142-7

ATP2B4 driven chromatin compaction exacerbates pancreatic cancer radiotherapy resistance