nejm.org

The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.

Brepocitinib, a first-in-class TYK2/JAK1 inhibitor, hit all primary and secondary endpoints in the largest-ever dermatomyositis trial, offering the first steroid-sparing treatment for a disease that has lacked targeted therapy for decades.

Gene-edited pancreatic islet cells transplanted into a patient with type 1 diabetes continue producing insulin 14 months later with no immunosuppressive drugs, advancing the case for a functional cure.

The FDA has delayed its decision on Eli Lilly's oral obesity pill orforglipron from March 28 to April 10, alongside delays for three other drugs in the new expedited review program.

MagicRNA's mRNA lipid nanoparticle therapy reprogrammed patients' own T cells inside the body, wiping out disease-driving B cells in refractory lupus patients without conventional cell manufacturing.

The PRIMA wireless retinal implant restored reading ability in 81 percent of blind AMD patients in a landmark NEJM trial — the first device to restore true form vision.

Enlicitide decanoate cut LDL cholesterol by 57% in 2,900+ patients in the CORALreef Lipids Phase 3 trial — matching injectable PCSK9 inhibitors in pill form, with no meaningful safety signals over 52 weeks.

The FDA's new Plausible Mechanism Pathway enables approval of one-patient gene editing therapies without traditional clinical trials, inspired by the first personalized CRISPR cure for an infant with a fatal metabolic disorder — but ethicists warn of a regulatory Pandora's box.