Blue­bird says EMA has be­gun re­view­ing its Lenti­Glo­bin gene ther­a­py; Sarep­ta bags rights to neu­ropa­thy gene ther­a­py

→ Blue­bird Bio says that its Eu­ro­pean ap­pli­ca­tion for Lenti­Glo­bin, its gene ther­a­py for trans­fu­sion-de­pen­dent β-tha­lassemia, has made it through the doors at the EMA. The ac­cep­tance sets up a mar­ket­ing de­ci­sion for this ther­a­py as the biotech plots a shift to com­mer­cial­iza­tion work.

→ Sarep­ta is all in on gene ther­a­pies. Bank­ing on ear­ly but no­table suc­cess­es for its Duchenne MD pro­gram, Sarep­ta is fol­low­ing up with its part­ners at Na­tion­wide Chil­dren’s Hos­pi­tal to gain rights to the gene ther­a­py can­di­date, neu­rotrophin 3, to treat Char­cot-Marie-Tooth neu­ropathies, in­clud­ing CMT type 1A.

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