explore -WSS

explore

author：focus 2025-09-28 00:52:27 - browse：75

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： Medicare details structure of new drug price negotiation program
Next article： Genentech weighs delaying cancer therapy after drug pricing law

focus

WSS

The future of health care in the House

Listen: Your guide to Wegovy's blockbuster heart study

Eli Lilly buys Point Biopharma in radiopharmaceuticals play

Timeline: Special counsel's investigation into Trump's handling of classified documents

An innovative path out of biotech’s “valley of death”

Presidential candidate assassinated in Ecuador, 1 suspect dead and 6 arrested

entertainment

explore

Bluebird and the case for gene therapy profits

knowledge

Psychedelics group wrestles with new pharma identity

Pharma budget tightening trickles down to Doximity

As demand for Covid shots wanes, Moderna seeks its next success

Target recalls 2.2 million additional candles due to laceration, burn hazards

BioMarin wins approval for gene therapy to treat hemophilia A

Chemotherapy drug shortage eases slightly but still widespread

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Analysis of telehealth claims data suggest service duplication

Duchenne breakthrough therapy leaves behind pioneering families

AI can decode, harness the power of the humane immune system

focus

Maui funeral service workers on devastation of fires: 'I'm in shock'

Histogen: Failed biotech offers rare public look inside

Senate panel eyes new effort on drug shortages

Some of the ways extreme heat will change life as we know it

Iran moves toward freeing 5 American citizens who had been imprisoned, in rare deal

Presidential candidate assassinated in Ecuador, 1 suspect dead and 6 arrested

hotspot

focus

hotspot

explore