Agios Pharmaceuticals, Inc.
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Latest From Agios Pharmaceuticals, Inc.
Plus deals involving Biocytogen/Radiance, Maruho/AOBiome, LGChem/Rhythm, IASO/Umoja, JW Therapeutics/2seventy and 13 other transactions.
The company hopes to position mitapivat as an oral treatment for patients across the thalassemia spectrum. A Phase III trial in non-transfusion-dependent alpha- or beta-thalassemia was positive.
The first gene editing medicine and gene therapy for sickle cell disease were approved as the ASH meeting got under way, but novel oral drugs may be key to making treatment more accessible.
The recent ASH meeting began with US FDA approvals of the first genetic medicines for sickle cell disease, but doctors and biopharma companies still see a need for new oral therapies and other options.
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