Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-025-04115-6A blueprint to accelerate rare pediatric gene therapy approvals

Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-026-04244-6In a phase 1 trial, the in vivo generation of anti-BCMA CAR-T cells by lentiviral delivery was feasible and did not lead to dose-limiting toxicities in five patients with relapsed or refractory multiple myeloma.

Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-026-04299-5Results of the phase 2 POLAR trial show that biomarker-guided treatment in patients with metastatic pancreatic cancer based on homologous repair deficiency leads to encouraging clinical response rates in immune cell-infiltrated tumors.

Nature Medicine, Published online: 24 March 2026; doi:10.1038/s41591-026-04262-4The first-in-human clinical trial of the LRRK2-targeting antisense oligonucleotide BIIB094 in Parkinson’s disease demonstrates that the treatment is well tolerated and produces dose-dependent reductions in cerebrospinal fluid levels of LRRK2 and phosphorylated Rab10, indicating successful target engagement.

Nature Medicine, Published online: 24 March 2026; doi:10.1038/s41591-026-04253-5In a large UK-based primary care randomized trial, immediate artificial intelligence (AI)-driven prioritization of chest X-rays did not significantly shorten time to computed tomography or lung cancer diagnosis versus standard workflow, suggesting that AI-based prioritization alone is unlikely to accelerate the lung cancer diagnostic pathway.