leisure time -WSS

leisure time

author：focus 2025-10-02 16:00:58 - browse：8721

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： BioMarin wins approval for gene therapy to treat hemophilia A
Next article： FDA gives detailed accounting of AI

explore

WSS

First Opinion Podcast: Long Covid needs a new approach

FDA warns J&J's Abiomed: We will regulate your software

NIH confirmation hearing shows politicization of research

Pfizer's RSV vaccine recommended for approval in Europe

STAT Summit: Michael J. Fox on progress in Parkinson's research

Pfizer's RSV vaccine recommended for approval in Europe

hotspot

leisure time

Bluebird and the case for gene therapy profits

knowledge

Jerome Adams on the need for new antivirals for Covid

Will Ozempic

What does generative AI mean for health care? We asked experts

What is artificial intelligence? Experts weigh in

BioMarin wins approval for gene therapy to treat hemophilia A

Dispute over threat of extinction posed by artificial intelligence looms over surging industry

How to save PrEP access — and even expand it

Impact of Dobbs ruling felt at reproductive medicine meeting

Virginia high school admissions case could be legal follow

It's time to take a new tack for treating Alzheimer's

knowledge

Mega Millions jackpot grows to $720 million ahead of Friday's drawing

STAT Summit: Michael J. Fox on progress in Parkinson's research

A new deal aims to ramp up use of AI in rehab care decisions

Eli Lilly expects to double planned employment in Boston center

Trump Organization, Michael Cohen agree to settle million

Reframing the conversation around gain

focus

explore

leisure time

entertainment