New CRISPR Therapeutics/Vertex Data Dangle The Hope Of Cure For SCD and TDT
With more data reported at ASH, Vertex and CRISPR Therapeutics added heft to the notion that functional cures are on the horizon for the inherited blood disorders sickle cell disease and beta-thalassemia. Attention is now turning to Bluebird’s marketing strategy after a filing delay.
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After the failure of a second candidate for alpha-1 antitrypsin deficiency, Vertex may have to put its cash reserves to use in M&A.
Vertex and partner CRISPR Therapeutics hope CDX001 can offer a single-use curative regimen for sickle cell disease and beta thalassemia patients. A regulatory filing is possible within 18-24 months.
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.