biotech

The Boston biotech closes a Series D round to advance ER-100, the first FDA-cleared therapy that uses partial epigenetic reprogramming to reverse cellular aging in humans.

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

A two-vector system inserts a CAR transgene at the TRAC locus inside the body, generating functional cancer-killing T cells without ex vivo manufacturing or lymphodepletion.

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.

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.