Sickle Cell Disease News

Agios to seek accelerated approval in US of oral mitapivat for SCD

After a meeting with the FDA, Agios Pharmaceuticals is planning to seek accelerated approval in the U.S. of mitapivat for ...

Beam Therapeutics Announces Publication of BEACON Phase 1/2 Data for risto-cel in Patients with Sickle Cell Disease (SCD) in The New England Journal of Medicine

Prestigious Publication Reinforces risto-cel’s Durable, Differentiated Clinical Data for the Treatment of SCD with Severe Vaso-occlusive Crises ...
Business Wire

bluebird bio Submits Biologics License Application (BLA) to FDA for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a ...

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for ...
Seeking Alpha

KIND Presents Preclinical Results of AND017 in Animal Models of Sickle Cell Disease (SCD), Myelodysplastic Syndromes (MDS), and β-Thalassemia at the 66th American Society of ...

SAN FRANCISCO, Dec. 12, 2024 /PRNewswire/ -- Kind Pharmaceutical ("Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC"), a clinical-stage biopharmaceutical company focused on developing ...
Business Wire

bluebird bio Announces FDA Priority Review of the Biologics License Application for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years ...

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for ...