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First gene therapy to treat rare blood disease nears European approval

“The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials.

“Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.

“Lentiglobin is Bluebird’s first gene therapy, and if approved, will be marketed under the brand name Zynteglo.

“There are dozens of biotech and pharma companies developing gene therapies for rare diseases today, but Bluebird was a pioneer. The first data from beta-thalassemia patients treated with lentiglobin were presented in 2014. Five years later, the gene therapy is on the cusp of approval.”

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