New Bluebird Data Hint at Gene Therapy’s Limits For Some Patients

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what it’ll need to do to get LentiGlobin to market. That includes running two additional studies with the goal of freeing patients from blood transfusions for 12 months. Leschly says that Bluebird’s overall regulatory strategy “very much still holds,” though there are now “some nuances” that the company will have to figure out and discuss with the FDA.

To treat beta-thalassemia patients with its LentiGlobin gene therapy, Bluebird harvests stem cells from a patient’s bone marrow and inserts into them a healthy version of the beta-globin gene. The healthy gene is carried into the cell by HIV viruses that have been genetically modified to be harmless. Once modified, the stem cells are grown in a culture and infused back into the patient in a one-time procedure.

The cells then head to the bone marrow and divide, giving rise to more cells with the correct gene—and therefore, eventually normal levels of hemoglobin. That’s the theory, at least.

Bluebird has been using this approach on beta-thalassemia, sickle cell disease, and childhood cerebral adrenoleukodystrophy, or CCALD. The first patient data from the CCALD study should come next year, Leschly says.

The firm went public with a splash in 2013, and even amidst biotech’s recent swoon, Bluebird’s shares as of Thursday afternoon were still worth more than five times their $17 IPO price. Thanks to its high valuation and positive drips and drabs of data released the past few years, Bluebird has been able to raise hundreds of millions of dollars in follow-on offerings—it had more than $900 million in the bank as of Sept. 30. The company has also expanded into fields such as gene editing and cellular immunotherapy for cancer. It’ll present preclinical data on a multiple myeloma immunotherapy partnered with Celgene at ASH.

“We’re just getting our mojo here, that’s our view,” Leschly says.

For more on Bluebird’s gene therapy, the data it’s amassed so far, and the hurdles to come, check out these stories. The company is holding a conference call this morning to discuss the data.

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