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.
Please update me of the approval and how to get it started.
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