A uniQure gene therapy for hemophilia B that has been under a clinical hold for four months can now resume testing. The hold was lifted after uniQure answered the FDA’s questions about whether the gene therapy caused the liver cancer diagnosed in one patient who received the therapy.
UniQure is making a case for lifting a clinical hold on its hemophilia B gene therapy, reporting Monday results of an independent investigation that appear to clear the experimental treatment as the cause of a patient’s cancer.
Founded by scientists behind the technology used in Johnson & Johnson’s Covid-19 vaccine, Leyden Laboratories is developing intranasal medicines intended to stop viruses before they infect the lungs. SARs-CoV-2 is one target, but the startup plans to address many respiratory pathogens.