Bluebird Bio Gets Relief, But Gene Therapy Hurdles Remain
Sickle Cell Patient’s AML Likely Unrelated To LentiGlobin
The jury is still out on the case of MDS and some remaining questions regarding the AML case may still hinder commercial uptake.
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Slated to begin a Phase III later this year of ADVM-022 in wet AMD, Adverum had to scuttle a Phase II study of the gene therapy in DME after a patient suffered ocular hypotension, panuveitis and vision loss in the treated eye.
Once the darling of biotech investors, Bluebird Bio’s go-it-alone strategy with Zynteglo has not paid off as German launch plans stall.
Building on Oxbryta, which is already approved in the US for the rare blood disorder, Global Blood Therapeutics has licensed two sickle cell disease programs from Sanofi.