Scientists have identified why the body's inflammatory response misfires in familial Mediterranean fever, a hereditary autoinflammatory disease that causes the immune system to overreact to minimal stimuli, triggering recurrent fever attacks.
UK regulators have authorized the INTERCEPT-Lynch Phase 1/2 trial of mRNA-4194, a preventive cancer vaccine developed by Moderna and the University of Oxford targeting people with Lynch syndrome, an inherited condition affecting 1 in 300 people with up to 80% lifetime cancer risk. The trial will begin dosing in summer 2026.
Cytokinetics announced its heart disease drug Myqorzo, already approved in one indication, succeeded in meeting twin efficacy goals in a Phase 3 trial for genetic heart disease, potentially boosting future sales.
The FDA has greenlit a first-of-its-kind gene therapy to treat a rare form of hereditary hearing loss, marking a breakthrough that could pave the way for other hearing impairment treatments. Follow-up research confirms the therapy yields lasting hearing gains for patients with inherited deafness.
International clinical trial results show that gene therapy for a rare form of genetic deafness successfully restored hearing in 90% of participants, with improvements lasting years after treatment. The study was co-led by Mass General Brigham and China's Eye & ENT Hospital of Fudan University.
