Cytokinetics announced detailed late-stage trial results showing its drug aficamten significantly improved symptoms and exercise capacity in patients with non-obstructive hypertrophic cardiomyopathy, a genetic heart condition with no approved treatments.
Medical experts specializing in Prader-Willi syndrome flagged serious adverse events in patients taking Neurocrine Biosciences' drug Vykat XR, raising safety concerns about the treatment for the rare genetic disorder.
Skylark Bio has dosed the first patient in its SONIX Phase 1/2 trial of SKY-GJB2, a gene therapy targeting GJB2-related hearing loss, the most common genetic cause of pediatric deafness. The trial follows earlier breakthrough gene therapy treatments that restored hearing in children born deaf.
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.
