Patients with beta-thalassemia benefit from Bluebird gene therapy

People with severe beta-thalassemia who live in Europe have been denied access to Bluebird Bio’s approved gene therapy after the company withdrew it from the market last April. European health systems balked at the nearly $2 million price tag for the gene therapy called Zynteglo, and when negotiations broke down, Bluebird walked away.

But Saturday, Bluebird relied largely on European patients enrolled in its beta-thalassemia clinical trials to demonstrate the successful, long-term durability of Zynteglo. The juxtaposition is awkward for Bluebird, showing business priorities don’t always align with the biotech’s ‘patients first’ mantra. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. What’s included? Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. This name will appear with your comment
https://www.statnews.com/2021/12/11/patients-with-beta-thalassemia-benefit-from-bluebird-gene-therapy-while-denied-access-in-europe/