Blue­bird lands an­oth­er rare dis­ease gene ther­a­py OK — but there are a few caveats to note

Rel­a­tive to the pro­tract­ed jour­ney of de­vel­op­ing a gene ther­a­py de­liv­ered by a lentivi­ral vec­tor, the speed at which blue­bird bio moved from hav­ing one ap­proved prod­uct to two was ex­treme­ly fast.

Ex­act­ly a month af­ter the FDA gave its green light to blue­bird’s Zyn­te­glo — to treat be­ta tha­lassemia — the agency came through with an ap­proval for a sec­ond gene ther­a­py for ear­ly, ac­tive cere­bral adrenoleukody­s­tro­phy (CALD). The drug, pre­vi­ous­ly known as eli-cel and now brand­ed Skysona, will have a whole­sale ac­qui­si­tion price of $3 mil­lion and should be­come avail­able by the end of this year.

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