rare-disease

Rocket Pharmaceuticals' Kresladi becomes the first gene therapy approved for LAD-I, an ultra-rare immune disorder that leaves children vulnerable to fatal infections.

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.

Precision BioSciences receives FDA Fast Track for PBGENE-DMD, a gene-editing therapy that excises faulty exons to restore near full-length dystrophin in up to 60 percent of Duchenne patients.

The FDA rejected uniQure's Phase I/II data for AMT-130 and now demands a sham surgery-controlled Phase 3 trial, reversing its own prior guidance and sending shares down 40 percent.

Beam Therapeutics' BEAM-302 has achieved the first-ever in-vivo base editing correction of a disease-causing mutation in human patients, reaching therapeutic AAT levels in a Phase 1/2 trial for alpha-1 antitrypsin deficiency. A pivotal Q1 2026 data readout is now imminent.

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.