biotech

Japan's health ministry has conditionally approved two iPS cell-derived drugs, marking the first time regenerative medicines built from induced pluripotent stem cells will reach patients commercially.

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.

A second-generation Pro-Active Genetics system spreads via bacterial conjugation to disable resistance genes on plasmids, working even inside biofilms.

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.