Gene-therapy leader bluebird bio (NASDAQ: BLUE) has made waves in the CAR-T space, although it's still trailing the CAR-T front-runners. But here are three things you might not know about the promising biotech.
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1. The company was originally named Genetix Pharmaceuticals
The name change occurred back in 2010 before the biotech went public, in conjunction with Nick Leschly being appointed the company's president and CEO.
Leschly, who goes by the title "chief bluebird," gave some background on the name change in a statement at the time:
"This name change exemplifies our intent to set a new, bold course for the future. The Eastern Bluebird is known to be a symbol of transition and renewal as well as a competitive and disciplined bird -- all traits that are well aligned with our culture and passion for transforming the lives of patients and their families."
The reason for the all-lowercase name is still a mystery.
Image source: Getty Images.
2. LentiGlobin isn't Bluebird's most advanced treatment
Most investors are rightfully focused on Bluebird's LentiGlobin gene therapy treatment. It offers nice potential in the orphan indication of beta thalassemia, plus a more lucrative indication in sickle-cell disease. But there's a gene-therapy product that Bluebird has been working on for longer than it's been developing LentiGlobin.
Its first gene-therapy drug, Lenti-D, treats cerebral adrenoleukodystrophy. The phase 2/3 Starbeam trial started in October 2013; the initial interim data looked good, with none of the patients having major functional disabilities and 16 of 17 patients having stabilization of their neurological function score, a measure of clinical deficits across 15 broad neurological functions such as stuttering, walking, and urinary incontinence. The full data from those patients is scheduled to be presented by the end of this year.
Bluebird recently announced that it reopened the trial to get experience manufacturing the product in Europe, and to boost the data package for its marketing applications in the U.S. and the EU.
3. There's more than just gene therapy and CAR-T
Bluebird's clinical-stage therapies are in the gene-therapy and CAR-T space, but it's working on a gene-editing technology that's still in the preclinical stage.
In 2014, the company bought privately-held Precision Genome Engineering. Much like CRISPR, which has dominated the news recently, Precision Genome's technology makes specific changes to patients' DNA to treat diseases, although it uses different enzymes called homing endonucleases and megaTALs. Bluebird believes these enzymes could be more efficient than CRISPR and other gene-editing technologies, because "they engage DNA repair pathways in a manner distinct from all other gene editing nucleases."
It could be a while before Bluebird has a gene-editing therapy in the clinic, so investors shouldn't be ascribing any value to the technology yet. But it's still worth keeping an eye on, for its potential to be a growth driver after the hoped-for launches of gene-therapy and CAR-T products.
A data-rich 2017
While Lenti-D and gene editing have potential to add incremental sales in the future, the best near-term opportunities are in LentiGlobin and CAR-T. Fortunately, there will be plenty of data released this year to help determine the therapies' potential.
In June, at the American Society of Clinical Oncology (ASCO) meeting, investors will get updated data for bb2121, Bluebird's lead CAR-T drug. And just a few days later at the European Hematology Association (EHA) meeting, Bluebird will present data for a trial using the company's new manufacturing procedure for LentiGlobin to treat beta thalassemia. Finally, in December, investors will get data on using the new LentiGlobin manufacturing procedure in treating patients with sickle-cell disease.
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