beta-thalassemia

Imara Inc (NASDAQ: IMRA) shares are falling after interim analyses of its Ardent Phase 2b trial of tovinontrine (IMR-687) in sickle cell disease (SCD) and Forte Phase 2b trial in beta-thalassemia.  Data from the sickle cell disease study interim analysis demonstrated that...

Bluebird Bio Inc (NASDAQ: BLUE) presented new results for betibeglogene autotemcel (beti-cel), investigational gene therapy for β-thalassemia (beta-thal) who require regular red blood cell (RBC) transfusions. New data demonstrate that adult and pediatric β-thalassemia...

The FDA has accepted Bluebird bio Inc's (NASDAQ: BLUE) marketing application for betibeglogene autotemcel (beti-cel) for β-thalassemia. The agency has granted priority review for Beti-cel, gene therapy for β-thalassemia across all genotypes who require regular red...

Bluebird Bio Inc (NASDAQ: BLUE) completed the rolling submission of its marketing application to the FDA seeking approval for betibeglogene autotemcel (beti-cel) gene therapy. The application covers adult, adolescent, and pediatric patients with β-thalassemia who require...

Vertex Pharmaceuticals Inc (NASDAQ: VRTX) and Arbor Biotechnologies have announced a new collaboration to develop ex vivo engineered cell therapies. The new agreement between Arbor and Vertex builds upon the companies' first partnership established in 2018.  Related:...

Barclays reiterated its Overweight rating on Acceleron Pharma Inc (NASDAQ: XLRN) after a meeting with management reinforced key elements of the firm's thesis on the company, particularly regarding the drug Luspatercept. Luspatercept And Acceleron's Recent Performance Acceleron's lead...