explore -WSS

explore

author：comprehensive 2025-09-27 22:47:31 - browse：66

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.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Walensky prepares to leave CDC — and Congress — behind
Next article： Is there an 'orca uprising'? Like orcas themselves, the answer is complex.

WSS

Vermont grapples with historic flooding as more rainstorms head for Northeast

Va. Republicans encourage early voting, a target of Trump's, ahead of fall elections

Women's World Cup brings attention to abuse in soccer

AMAs digital advisors table RPM coding reform proposal, again

Study gives hint on South Asians’ higher risk of cardiovascular disease

Watch the official trailer for 'Ahsoka' series

hotspot

explore

Bluebird and the case for gene therapy profits

hotspot

Moonlake's readout produced a cash windfall. Risks remain

Too many hospitalized Covid

The high cost of giving birth even with insurance

Tornado touches down near Chicago's O'Hare airport amid extreme weather nationwide

Medical records are filled with copy

China sends large group of warplanes, navy ships towards Taiwan in forceful display

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

Britney Spears to release memoir, 'The Woman In Me,' later this year

Dobbs anniversary: the lost opportunity of abortion as health care

Britney Spears to release memoir, 'The Woman In Me,' later this year

focus

Bernie Sanders gets little from pharma CEOs on drug prices

Russia's threat to pull out of Ukraine grain deal raises fears about global food security

Britney Spears to release memoir, 'The Woman In Me,' later this year

Iran's president begins a rare visit to Africa 'to promote economic diplomacy'

Russia's threat to pull out of Ukraine grain deal raises fears about global food security

Iran's president begins a rare visit to Africa 'to promote economic diplomacy'

knowledge

entertainment

focus

leisure time