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Cambridge, Mass. – Rare disease company Agios Pharmaceuticals reached the primary endpoint in a Phase III study for its drug pyrukynd (mitapivat) in adults with non-transfusion-dependent alpha- or beta-thalassemia, according to the company’s announcement on Wednesday The Phase III ENERGIZE study met the primary endpoint of hemoglobin response, defined as...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced it will sponsor Citizen Scientist: Unpacking the Science of Sickle Cell Disease, an initiative developed by the Sickle Cell Community Consortium (SCCC) to promote health...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced that it has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for TIBSOVO® (ivosidenib tablets) as a potential...
Cambridge — Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, announced that the company is scheduled to present at the following March investor conferences: Cowen 43rd Annual Health Care Conference on Monday, March 6, 2023 at 1:30 p.m. ET; and...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has initiated plans to lease a build-to-suit 21,000 square foot current Good Manufacturing Practices (cGMP)...
GAINESVILLE, Fla., and CAMBRIDGE, Mass., – Applied Genetics Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, which include X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM), announces two important initiatives to help patients with potential interest...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced the appointment of Hope D’Oyley-Gay as General Counsel. In this role Ms. D’Oyley-Gay will oversee legal, IP and compliance...