Multiple undisclosed preclinical programs
Undisclosed rare diseases
PreclinicalActive discovery
Key Facts
Indication
Undisclosed rare diseases
Phase
Preclinical
Status
Active discovery
Company
About Agios Pharmaceuticals
Founded in 2008 and headquartered in Cambridge, Massachusetts, Agios Pharmaceuticals has evolved from a pioneering cellular metabolism research company into a commercial-stage rare disease leader. The company's lead product, PYRUKYND® (mitapivat), is FDA-approved for pyruvate kinase (PK) deficiency and is being evaluated in late-stage trials for thalassemia and sickle cell disease. Following the strategic divestiture of its oncology portfolio to Servier for up to $2 billion, Agios is singularly focused on building a leading rare disease franchise rooted in PK activation and cellular metabolism science, targeting hemolytic anemias and other rare conditions.
View full company profileTherapeutic Areas
Other Undisclosed rare diseases Drugs
| Drug | Company | Phase |
|---|---|---|
| JOTROL™ | Jupiter Neurosciences | Clinical |
| Rare Disease Drug Discovery Program | RevolKa | Preclinical |
| rAAV-based Gene Therapy Programs | Takara Bio | Discovery / Preclinical |