Alpha and beta thalassemia affect different parts of the hemoglobin protein. Both conditions can cause anemia ranging from mild to severe, which may require blood transfusions to treat.

These data validate the further development of Editas’ proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia,” said Linda C ...

Revolutionary advancements in blood cancer treatment, from targeted therapy to gene editing, promise more effective and ...

CAMBRIDGE, Mass., May 28, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare ...

--Agios Pharmaceuticals, Inc., a leader in cellular metabolism and pyruvate kinase activation pioneering therapies for rare diseases, today announced that its management team is scheduled to ...

Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical ...

Vertex accesses Orna’s proprietary LNPs to co-develop in vivo gene-editing therapies for sickle cell disease and beta-thalassemia, potentially leading to an improved version of Casgevy $635 ...

Thalassemia types are classified based on which part of the hemoglobin molecule is affected—alpha or beta—and the severity of the condition, described as trait, intermedia, or major. Hemoglobin, ...

Dottie Caplan, who previously served as Senior Vice President, Patient Advocacy and Engagement, will be responsible for leading Applied’s patient engagement and advocacy efforts as well as the ...

Alpha thalassemia is a blood disorder in which the body has a problem producing alpha globin, a component of hemoglobin, ... The imbalance in alpha and beta globin causes anemia and leads to the other ...