Long focused on antioxidants, researchers are now exploring a new route to combat prostate cancer: pro-oxidants.

GNT-0003 is under development for the treatment of crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV (adeno-associated virus) ...

Other AAV-based gene therapies have also run into problems with liver side effects, including Astellas' X-linked myotubular myopathy therapy AT132, which has been linked to four deaths.

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

kids.wustl.edu OBJECTIVE To evaluate muscle pathology and clinical characteristics in patients with a myopathy and serum antibodies to the Jo-1 antigen (histidyl t-RNA synthetase). BACKGROUND ...