Researchers have developed a new interventional treatment for hepatic myelopathy, a rare neurological complication of chronic liver disease that previously could only be cured through liver transplantation.
The FDA has approved Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel) for patients aged two years and older with sickle cell disease and transfusion-dependent beta thalassemia, expanding from the previous minimum age of 12 years. The approval was granted under the agency's Commissioner's National Priority Voucher program.
Texas-based biotech Ollin Biosciences secured $330 million in funding to advance late-stage clinical studies of its experimental eye disease treatment, representing one of the largest recent funding rounds in ophthalmology.
The 2026 Breakthrough Prize honored six laureates with $3 million awards for discoveries including gene therapies for inherited blindness and sickle cell disease, as well as groundbreaking work on KRAS cancer targets. Boston-area researchers were among those recognized.
