Key question facing new gene therapy for blood disorders: How long will it last?

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sickle cell

New versions of Bluebird Bio’s gene therapy for inherited blood disorders yielded significant benefits for patients, according to updated results from ongoing clinical trials released [June 15]. But whether those benefits will endure for the patients with those conditions, beta-thalassemia and sickle cell disease, remains an open question.

Here’s a rundown:

Bluebird has now treated six sickle cell patients with the new version of LentiGlobin. Among other changes, the new version is derived from stem cells harvested more easily from their blood rather than bone marrow, as was the case with the old version. No serious safety problems have surfaced.

LentiGlobin works by producing healthy, anti-sickling hemoglobin, that, in turn, replaces damaged, sickled red blood cells known to cause anemia and extremely painful sickle cell “crises” in patients.

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Four of the sickle cell patients have been followed for longer than three months and are now producing anti-sickling hemoglobin.

In a [June 14] interview, Bluebird’s chief medical officer, David Davidson, said the LentiGlobin data being presented this weekend are starting to show convincing evidence that the gene therapy can transform patients with two gene mutations — meaning they have active sickle cell disease — into what is known as “trait” patients: those who only carry a single mutation and are therefore asymptomatic and live normal lives.

[Original article is behind paywall.]

Read full, original post: New Bluebird data show promising benefits for gene therapy, if they last

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