NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. today announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder. The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with...
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NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has resubmitted its Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) to the U.S. Food and Drug Administration (FDA or...
AMHERST, Mass. – Researchers at the University of Massachusetts Amherst recently released a first-of-its-kind study that focuses on the rare autoimmune disorder aplastic anemia to understand how a subset of cells might be trained to correct the overzealous immune response that can lead to fatal autoimmune disorders. The research, published...
Worcester, Mass. – A study led by UMass Chan Medical School viral immunologists Liisa Selin, MD, PhD, and Anna Gil, PhD, discovered similarities in immune system dysfunction as a potential biomarker among people living with long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). The research also introduced a novel treatment...
The increasing amount of patients with obesity and type 2 diabetes benefit greatly from the recently developed GIPR:GLP-1R co-agonists. These novel compounds lead to substantial weight loss, offering a revolutionary approach to patients worldwide. Although the hormone glucose-dependent insulinotropic polypeptide (GIP) was already shown by Helmholtz Munich scientists to decrease...
MINNEAPOLIS – OX2 Therapeutics, Inc., a privately held Minneapolis based company, announced today encouraging early results from phase 1 human trials of their new cancer treatment. Developed to combat recurrent high-grade brain tumors, the treatment utilizes a newly developed peptide inhibitor. “OX2 works to explore the therapeutic potential of the new immune...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease, a rare, inherited neurodegenerative disorder...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for...
LEXINGTON, Mass. and AMSTERDAM, Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease....
LEXINGTON, Mass. and AMSTERDAM, the Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary endpoint,...