Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals is a biopharmaceutical company dedicated to developing therapies for rare diseases, with a focus on cellular metabolism. They are committed to building connections with patients, partners, and colleagues to create life-changing therapies. Their mission emphasizes scientific innovation, patient-centricity, and social responsibility, aiming to improve lives through groundbreaking medicines for unmet medical needs.
Industries
Nr. of Employees
medium (51-250)
Agios Pharmaceuticals, Inc.
88 Sidney Street, Cambridge, MA 02139-4169
Patents
Compounds and methods useful for stabilizing phenylalanine hydroxylase mutations
US-11919915-B2
View Details
Compounds and methods useful for stabilizing phenylalanine hydroxylase mutations
US-11919915-B2
View DetailsProducts
Approved oral pyruvate kinase activator for pyruvate kinase deficiency
An orally administered pyruvate kinase activator approved for treatment of adults with pyruvate kinase deficiency in multiple jurisdictions, used to reduce hemolysis and improve hematologic parameters.
Clinical-stage pyruvate kinase activator programs
Late-stage and early-stage clinical development programs evaluating oral pyruvate kinase activators across indications including thalassemia, sickle cell disease and lower-risk myelodysplastic syndromes.
Approved oral pyruvate kinase activator for pyruvate kinase deficiency
An orally administered pyruvate kinase activator approved for treatment of adults with pyruvate kinase deficiency in multiple jurisdictions, used to reduce hemolysis and improve hematologic parameters.
Clinical-stage pyruvate kinase activator programs
Late-stage and early-stage clinical development programs evaluating oral pyruvate kinase activators across indications including thalassemia, sickle cell disease and lower-risk myelodysplastic syndromes.
Services
Single point of engagement offering disease education, community connections, prescription fulfillment, insurance/funding assistance and dedicated patient support managers for eligible patients.
Pathway for healthcare providers in selected countries to request access to investigational or approved therapies where commercial availability is limited.
Formal data request process and form to enable qualified external requests for clinical trial data and associated materials.
Single point of engagement offering disease education, community connections, prescription fulfillment, insurance/funding assistance and dedicated patient support managers for eligible patients.
Pathway for healthcare providers in selected countries to request access to investigational or approved therapies where commercial availability is limited.
Formal data request process and form to enable qualified external requests for clinical trial data and associated materials.
Expertise Areas
- Cellular metabolism and enzyme modulation (pyruvate kinase activation)
- Rare hematologic disorders (pyruvate kinase deficiency, thalassemia, sickle cell disease, lower-risk MDS)
- Clinical development and trial execution for rare diseases
- Translational research and biomarker discovery
Key Technologies
- Pyruvate kinase activation (small-molecule activators)
- Preclinical rodent disease models
- Ex vivo red blood cell functional assays
- Metabolomics and multi-omics platforms
News & Updates
First rare disease medicine approved by Agios in the U.S., EU, and Great Britain.
First rare disease medicine approved by Agios in the U.S., EU, and Great Britain.