Sep 30, 2026
Ultragenyx Pharmaceuticals' apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The failure of what was once considered a potential blockbuster therapy is forcing Ultragenyx to examine significant cost cuts.
The FDA extended the review period for Capricor Therapeutics' experimental cell therapy deramiocel for Duchenne muscular dystrophy by three months after the company submitted new data with a refined indication.
The FDA halted testing of Regenxbio's gene therapy for Hunter Syndrome after spinal MRI scans detected abnormalities in five trial participants. This is the second clinical hold placed on a Regenxbio gene therapy program.
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.
The European Medicines Agency recommended withdrawing marketing authorization for Amgen's rare autoimmune disease drug Tavneos, citing allegedly incorrect and misleading data used in the approval process and lack of benefits outweighing risks.
The FDA indicated it would reverse its earlier rejection of Regenxbio's gene therapy for a deadly childhood disease, stating that existing data would be sufficient to support an accelerated approval bid. The reversal represents a significant policy shift.
Agios Pharmaceuticals announced it will stop developing its experimental drug for a form of blood cancer after a mid-stage trial failed to show sufficient benefit, marking a setback for the rare disease program.
Five years after a disastrous trial where some boys died while others had astonishing recoveries, Astellas is returning with a revised gene therapy treatment for X-linked myotubular myopathy (XLMTM), offering new hope to a devastated rare disease community.
Entrada Therapeutics' next-generation therapy for Duchenne muscular dystrophy fell short in an early clinical study, representing a setback as multiple companies race to develop improved treatments for the genetic muscle-wasting disease.
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.
Kyverna Therapeutics announced plans to submit its one-time CAR-T cell therapy for stiff person syndrome to the FDA after late-stage trial showed improvements in mobility and reduced disabilities in patients with the rare neurological disease.
Scientists at the Buck Institute for Research on Aging demonstrated that an orally administered small molecule, N-propargylglycine (N-PPG), can completely prevent the formation of calcium oxalate kidney stones in preclinical studies.