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New CRISPR Therapeutics/Vertex Data Dangle The Hope Of Cure For SCD and TDT

 
• By Alexandra Shimmings

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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• Source: Shutterstock

bluebird bio will need to capitalize on its leader position if it is to maintain first-mover advantage over CRISPR Therapeutics AG/Vertex Pharmaceuticals Incorporated in a potential blockbuster market for genetic treatments for sickle cell disease (SCD) and beta-thalassemia.

The companies have each presented data from more patients for their respective products, LentiGlobin for Sickle Cell Disease, betibeglogene autotemcel (beti-cel) and CTX001, all one-time genetic treaments for SCD and beta-thalassemia, at the American Society of Hematology meeting

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