Bluebird lands another rare disease gene therapy OK — but there are a few caveats to note

Amber Tong

Senior Editor

Relative to the protracted journey of developing a gene therapy delivered by a lentiviral vector, the speed at which bluebird bio moved from having one approved product to two was extremely fast.

Exactly a month after the FDA gave its green light to bluebird’s Zynteglo — to treat beta thalassemia — the agency came through with an approval for a second gene therapy for early, active cerebral adrenoleukodystrophy (CALD). The drug, previously known as eli-cel and now branded Skysona, will have a wholesale acquisition price of $3 million and should become available by the end of this year.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

BECOME A PREMIUM SUBSCRIBER

Bluebird lands another rare disease gene therapy OK — but there are a few caveats to note

Amber Tong

Senior Editor

Unlock this article instantly by becoming a free subscriber.

Head of Regulated Labs Market Strategy

Benchling

San Francisco, CA, USA

The next generation of AI biotechs is here. Can they deliver on the hype?

AbbVie beats first-quarter expectations, but Humira guidance leaves analysts confused

Bristol Myers to shutter Bay Area cell therapy R&D facility as it eliminates jobs and costs

UCB makes major management changes; Bristol Myers’ ADC partner tops list of new CEOs

Updated: FDA approves Pfizer’s hemophilia B gene therapy, with $3.5M price tag

Ophthalmic biotech raises $16.5M; Pfizer’s AI collaboration bears fruit

Blue­bird lands an­oth­er rare dis­ease gene ther­a­py OK — but there are a few caveats to note

Senior Editor

Unlock this article instantly by becoming a free subscriber.

more like this

Benchling

San Francisco, CA, USA

Bluebird lands another rare disease gene therapy OK — but there are a few caveats to note