explore -WSS

explore

author：focus 2025-09-03 07:51:39 - browse：5391

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： Guidelines to prevent youth baseball injuries need more muscle
Next article： MultiPlan and major insurers sued for alleged medical price

hotspot

leisure time

WSS

Apple Vision Pro: A first look at health apps on the new VR headset

FDA drug safety: Singulair case shows systemic surveillance flaws

Cholera vaccine supply gets boost as demand surges globally

Covid misinformation case heads to Supreme Court

Novo Nordisk to expand supply of Wegovy for U.S. patients

Congressmen charge Covid hearing ‘could have been an email’

leisure time

explore

Bluebird and the case for gene therapy profits

leisure time

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

Close Medicare's dangerous gaps in addiction treatment coverage

Walensky prepares to leave CDC — and Congress — behind

MassMutual is introducing free genetic testing for members

Bright Health sells Medicare Advantage business to Molina

Some dementia cases could be undiagnosed liver disease

ChatGPT in medicine: STAT answers readers' burning questions

Juul spent big to court Black leaders to promote its e

Trump demands the U.S. pay no more for drugs than other countries … again

New Ultima Genomics DNA sequencer can read genome for $100

Wikipedia

Element Biosciences CEO lays out genome sequencing strategy

Covid misinformation case heads to Supreme Court

New Ultima Genomics DNA sequencer can read genome for $100

A $475,000 price tag for a new cancer drug: crazy or meh?

What we need to know about Arena Bioworks

Key Senate committee drafts bipartisan bill to stem drug shortages

entertainment

hotspot

leisure time

Wikipedia