What happened

Bluebird Bio (BLUE -6.25%) stock got off to a roaring start on Monday, trouncing the S&P 500 index with a nearly 8% gain. The market was cheered by a clinical update and the potential for a new regulatory application in the near future.

So what

On Saturday at a healthcare conference, Bluebird presented recent data on its gene therapies for beta-thalassemia and sickle cell disease (SCD).

The former, Zynteglo, produced transfusion independence for 41 participants in the 63-person set of phase 3 clinical trials. Beta thalassemia is an inherited blood disorder in which the body produces less hemoglobin than usual. Additionally, testimonials from three years after treatment indicate that 93% of those recipients were able to work, well above the 67% baseline. All participants reported overall improvement from the medication.

Zynteglo received Food and Drug Administration (FDA) approval in April. It was the first gene therapy green-lighted for beta-thalassemia patients requiring regular red blood cell transfusions. 

Meanwhile, there was good news about the other (and related) Bluebird gene therapy that was a subject of the presentation. The biotech reported it is on track to submit lovo-cel, an investigational treatment that targets SCD, in the first quarter of next year for a Biologics License Application (BLA) from the FDA.

Now what

Despite some hiccups with lovo-cel (previously known as LentiGlobin), Bluebird has had quite a bit of success in 2022. It earned not one but two FDA approvals, that for Zynteglo plus a nod for Skysona, a treatment for neurological cerebral adrenoleukodystrophy. It seems the company is well positioned to keep that momentum going, particularly with the lovo-cel program steaming ahead.