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ARMR Sciences begins Phase 1 trials of a vaccine that trains the immune system to intercept fentanyl before it reaches the brain, a preventive approach with no precedent in addiction medicine.
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.
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.