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...
treatment News
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced positive results from its Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent . Data from the study will be featured in an oral...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, announced that it has submitted a New Drug Application (NDA) for mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults...
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...
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 retinal diseases, today reported 12-month data from its ongoing achromatopsia (ACHM) Phase 1/2 clinical trials, including data from all adult...
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 retinal diseases, today reported additional positive data from the ongoing X-linked retinitis pigmentosa (XLRP) Phase 1/2 clinical trial, including 12-month...
Changchun, China – Hepatocellular carcinoma (HCC) is the most common type of liver cancer. It is a major global health concern with rising rates, particularly in North Africa and East Asia. Although potentially curable in the early stages, HCC often remains undetected until it’s too late for effective treatment. This...
Jyväskylän, Finland – Researchers from the University of Jyväskylä in collaboration with the Institute of Biomedicine University of Turku and Nova Central Finland have developed an artificial intelligence tool for automatic colorectal cancer tissue analysis. The refined neural network outperformed all previous solutions in the task. The neural network offers...
Palo Alto, Calif. – Using novel machine learning tools developed at Stanford Medicine, researchers have mapped three distinct cellular configurations that correspond to clinical outcomes for patients with a rare, difficult-to-treat cancer called soft tissue sarcoma. In particular, the technique identified a cellular neighborhood that correlated with a positive response...