Changing the Treatment Paradigm for Pyruvate Kinase Deficiency with Lentiviral-Mediated Gene Therapy: Interim Results From an Ongoing Global Phase 1 Study The poster presentation includes data ...

CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today ...

Rocket's clinical programs using lentiviral vector (LV) based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and ...

CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined ...

– Data Suggest Long-term Treatment with PYRUKYND ® in Adults with PK Deficiency is Associated with Improvements in Hemoglobin, Iron Overload, Transfusion Burden and Patient-reported Outcomes, ...

CAMBRIDGE, Mass., Feb. 17, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, ...

Agios Pharmaceuticals Inc.’s fourth-quarter and full-year 2023 financial report in mid-February highlighted further prospects with Pyrukynd (mitapivat), the pyruvate kinase activator for hemolytic ...

Pyrukynd (mitapivat) is a prescription drug that’s used to treat hemolytic anemia. Pyrukynd comes as an oral tablet. Pyrukynd is used to treat hemolytic anemia in adults who have pyruvate kinase (PK) ...