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author：knowledge 2025-09-30 00:47:55 - browse：88

Adam's take main illustration — Molly Ferguson/STAT

Happy Groundhog Day eve. Some takes and thoughts on Bluebird Bio’s quest for gene therapy profits, a reality check on Vertex’s highly touted pain drug, and more ADC investor FOMO.

Bluebird and the case for gene therapy profits

Genetic treatments that are potential cures for inherited diseases are a huge medical advance, but can these transformational (and expensive) products become a profitable business? I’m not sure anyone knows yet for sure, but pay attention to Bluebird Bio over the next year for some clues.

Bluebird is unique among the biotech and pharma companies selling gene therapies (or CRISPR-based medicines) today because it has no other marketed products. With the recent approval of Lyfgenia for sickle cell disease (cost: $3.1 million), Bluebird sells three gene therapies. The other products are Zynteglo for beta thalassemia ($2.8 million) and Skysona for cerebral adrenoleukodystrophy ($3 million). It’s a pure-play commercial gene therapy business. It’s not even trying to develop additional gene therapies currently. R&D expenses are tied to gathering long-term data on its existing products.

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