Blue­bird goes 2-for-2 on gene ther­a­pies at FDA ad­comm with thumbs up for beti-cel

For the sec­ond straight day, the FDA’s Cel­lu­lar, Tis­sue and Gene Ther­a­pies ad­comm vot­ed unan­i­mous­ly in fa­vor of FDA ap­prov­ing a blue­bird bio gene ther­a­py, this time by a 13-0 vote in fa­vor of beti-cel as a po­ten­tial treat­ment for a blood dis­or­der known as β-tha­lassemia for those who re­quire reg­u­lar blood trans­fu­sions.

The sec­ond straight unan­i­mous thumbs up opens the po­ten­tial for two FDA ap­provals lat­er this sum­mer for blue­bird — al­though the agency on Fri­day raised some man­u­fac­tur­ing con­cerns for both ther­a­pies.

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