Tag Archives: gene

Bluebird CEO defends $1.8 million gene therapy price — ‘It’s really thinking about it differently’

Bluebird Bio CEO Nick Leschly on Friday defended the biotech company’s $ 1.8 million price tag for its new gene therapy to treat a rare genetic blood disorder. Bluebird’s therapy, Zynteglo, was approved in Europe earlier this month for patients with beta thalassemia who require regular blood transfusions to manage their disease and have no… Read More »

Three years after undergoing gene therapy, his ‘last shot’ hit the target

Brenden was the first patient enrolled in what became a nine-patient, four-center trial that also involved patients at the National Institutes of Health (NIH) in Maryland, the University of California at Los Angeles, and Great Ormond Street Hospital for Children in London. The trial, funded by the NIH and the California Institute for Regenerative Medicine,… Read More »