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 ...

Agios Pharmaceuticals is down some 40% since I last looked at this commercial stage biotech. Read the report to find updates on my thesis on the AGIO stock.

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, ...

CRISPR Therapeutics CRSP and Intellia Therapeutics NTLA are leading developers of therapies that utilize the Nobel ...