Gene Therapy
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FDA Reverses Course on UniQure's Huntington's Gene Therapy, Demanding Sham Surgery Trial in Setback That Could Add Years to Approval Timeline
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.
Base Editing Corrects a Genetic Disease for the First Time in Living Patients, Putting BEAM-302 on Track for FDA Accelerated Approval
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.
UC San Diego Team Builds a CRISPR Gene Drive That Spreads Through Bacteria and Strips Away Antibiotic Resistance
A second-generation Pro-Active Genetics system spreads via bacterial conjugation to disable resistance genes on plasmids, working even inside biofilms.
The FDA's Plausible Mechanism Pathway: How Baby KJ's Personalized CRISPR Therapy Is Rewriting the Rules of Drug Approval
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.
Epigenetic CRISPR Technique Reactivates Silenced Genes Without Cutting DNA
UNSW and St. Jude researchers demonstrate that removing methyl tags from DNA can switch genes back on, opening safer paths for treating sickle cell disease.