Salt Lake City, Utah – Some families call it a trial of faith. Others just call it a curse. The progressive neurological disease known as spinocerebellar ataxia 4 (SCA4) is a rare condition, but its effects on patients and their families can be severe. For most people, the first sign...
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MILAN, Italy – With the decision by the U.S. Food and Drug Administration and the European Commission to grant market authorization for Waskyra™, a gene therapy to treat Wiskott-Aldrich syndrome, AGC Biologics’ Milan Cell and Gene Center of Excellence announced it will continue to support the commercial development of this vital,...
Baltimore, Maryland – Age-related changes that cause the skin to stiffen and become less elastic may also contribute to higher rates of metastatic skin cancer in older people, according to research by investigators from the Johns Hopkins Kimmel Cancer Center. The study, published March 12 in Nature Aging, shows that increased...
Study shows botensilimab “switches on” the body’s own T-cells to attack a common, treatment-resistant form of colorectal cancer LEXINGTON, Mass. — Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology, today presented new translational data at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. The study demonstrates that botensilimab-based...
LEXINGTON, Mass. — Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology innovation, today announced the publication of clinical results from the ovarian cancer cohort of its Phase 1b C-800-01 trial evaluating botensilimab plus balstilimab (BOT+BAL) in The Journal for ImmunoTherapy of Cancer (JITC). The peer-reviewed manuscript titled, “Botensilimab (Fc-enhanced anti–CTLA-4 antibody) plus balstilimab...
CAMBRIDGE, Mass. — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced its anticipated key 2025 milestones and value-driving catalysts through 2026. “2024 was marked by exceptional progress at Agios. We delivered on all our key priorities,...
CAMBRIDGE, Mass. — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced topline results from the 52-week double-blind period of the global RISE UP Phase 3 trial of mitapivat, an oral pyruvate kinase (PK) activator, in patients aged...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported a full analysis of updated data, including patient-reported outcome (PRO) measures, from its global Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase...
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 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...