First hepatitis delta treatment wins FDA approval
The US Food and Drug Administration approved the first treatment specifically indicated for chronic hepatitis delta virus (HDV) infection, the most aggressive form of viral hepatitis. The drug, a once-daily oral entry inhibitor developed by a German biotech firm, reduced HDV RNA levels to undetectable in 44% of patients in a Phase 3 trial published in the New England Journal of Medicine. HDV affects people already infected with hepatitis B and rapidly progresses to cirrhosis and liver cancer if untreated.
Oral GLP-1 shows promise for obesity without injections
Positive Phase 2 results for a new oral GLP-1 receptor agonist were published in JAMA on Wednesday, showing 15.4% average weight loss over 36 weeks compared to 2.3% for placebo. The drug, taken as a daily pill rather than injected, could offer an alternative for patients unwilling or unable to use injectable therapies like semaglutide and tirzepatide. Gastrointestinal side effects were reported in 38% of participants but were mostly mild to moderate. Phase 3 trials are expected to begin in early 2027.
CRISPR gene therapy reaches new milestone in infants
Scientists at the Innovative Genomics Institute reported a milestone in personalized CRISPR medicine: an infant with carbamoyl phosphate synthetase I (CPS1) deficiency — a rare urea cycle disorder — received a custom-designed in vivo CRISPR therapy within six months of diagnosis. The treatment, delivered via lipid nanoparticle infusion, dramatically reduced the infant's need for ammonia-scavenging medication and showed no adverse effects after three months of follow-up.