Bluebird backing boosts beleaguered gene therapy space

The unanimous backing of Bluebird's gene therapies by FDA panels last week not only throws that company a lifeline, it also gives a boost to a section of the biotech world that has taken a particularly big hit to sentiment. True, final approvals of the lentiviral projects are still pending, and reimbursement will be another issue, but comments at the hearings suggest that US physicians and patients are open to using gene therapies despite ongoing safety concerns, largely around the risk of cancer, and questions about lack of durability. For the first project, eli-cel, for a very rare childhood condition, lack of other options for those without a matched donor for stem cell transplant helped win the risk-benefit argument. A green light for beti-cel for beta thalassaemia also looks likely after the project impressed on durability, with some patients transfusion free after four years. Bluebird shares opened up 40%, but the company remains in dire financial straits. The market for eli-cel is tiny, and competition is coming behind for beti-cel. Approvals and the swift sale of subsequent priority review vouchers must happen without delay for these gene therapy stories to have a chance of a happy ending.

Bluebird's days of reckoning: the outcomes   
Project Vote Next step 2028e sales  
Beti-cel (beta-thalassaemia gene therapy) 13-0 in favour of benefit over risks FDA decision due by Aug 19  $120m*
Eli-cel (CALD gene therapy) 15-0 in favour of benefit over risks FDA decision due by Sep 16  $21m
Note: *beta-thalassaemia sales only; CALD=cerebral adrenoleukodystrophy. Source: company communications.

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