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Fewer Transfusions Needed

In a new study, gene therapy appears promising to treat the blood disorder β-thalassemia, NPR reports. It adds that the treatment led patients to need fewer blood transfusions and had few side effects.

An international team of researchers conducted two phase 1-2 studies to examine the safety and efficacy of their β-thalassemia gene therapy in patients, as the report in the New England Journal of Medicine. Their therapy involved removing the patients' own red blood cells and editing them using the LentiGlobin BB305 vector, which encodes adult hemoglobin with a T87Q amino acid substitution. Of the 22 patients treated, nine had the severe β00genotype.

By about 26 months after treatment, 12 of the 13 patients with non-β00genotypes had stopped receiving red-cell transfusions, the researchers report, and three of the nine patients with the β00genotype were able to stop transfusions, while the rest needed them less often. At the same time, the researchers report no severe adverse effects.

"β-thalassemia is one of the first examples in which gene therapy could be applied to a large population of patients who reside mostly in developing countries," writes Harvard Medical School's Alessandra Biffi in a related editorial in NEJM.

NPR notes that the recently approved gene therapy Luxturna for retinal dystrophy, though, has a hefty price tag of $425,000 per eye. It adds the developer of this β-thalassemia treatment, Bluebird Bio, hasn't yet announced its cost.

The company's chief medical officer, David Davidson, tells Technology Review that the firm plans to seek approval for the therapy in Europe later this year and after that in the US.