Lonza and Genetix Biotherapeutics agreed that Lonza will expand capacity to support the manufacture of Genetix’s ZYNTEGLO™, the only FDA-approved gene therapy for pediatric and adult patients with ...

Thalassemia is a group of anemias that result from inherited defects in the production of the beta chain of hemoglobin. It is stabilized by gamma globin, which combines to form fetal hemoglobin. One ...

Researchers will investigate the long-term effects of sickle cell disease, beta thalassemia and acute leukemias by linking records across England and surveying patients, as part of the Haematology ...

The FDA delivered a pair of holiday season approvals a week apart, signing off on Agios’ oral medicine Aqvesme for the treatment of anemia in adults with alpha- or beta-thalassemia on Dec. 23, and ...

Aqvesme is expected to be available in late January 2026. The Food and Drug Administration (FDA) has approved Aqvesme™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia ...

Good day, and welcome to Agios Pharmaceuticals' Investor Conference Call and Webcast. [Operator Instructions] Please be advised that this call is being recorded at Agios' request. I would now like to ...

The U.S. Food and Drug Administration (FDA) on Tuesday approved Agios Pharmaceuticals, Inc.’s (NASDAQ: AGIO) Aqvesme (mitapivat), an oral pyruvate kinase (PK) activator, for anemia in adults with ...

Company will host investor conference call and webcast tomorrow, December 24 at 8:00 a.m. ET CAMBRIDGE, Mass., Dec. 23, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO), a commercial-stage ...

What Is Aqvesme, and Why Does It Matter? Aqvesme (mitapivat) is a new oral medicine approved by the FDA for treating anemia in adults with alpha- or beta-thalassemia, including those who need and ...

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...