uniQure Announces Clinical Update on Hemophilia B Gene Therapy Program
Company to hold conference call today at 8:30 a.m. ET
LEXINGTON, Mass. and AMSTERDAM, Dec. 21, 2020 (GLOBE NEWSWIRE) uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that its hemophilia B gene therapy program, including the pivotal, Phase III HOPE-B study, has been placed on clinical hold by the U.S. Food and Drug Administration (FDA). Patient dosing is complete in each of uniQure’s three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.
investigators presented noteworthy findings on an innovative gene therapy for sickle cell disease, CAR T-cell therapy in high-risk large B cell lymphoma, and research in stem cell transplantation.
In this in-between-isode of MedPage Today s Podcast series, Anamnesis, one of our reporters discusses some of these data.
Host: Hello, and thanks for joining us for the latest in-between-isode of Anamnesis.
ASH 2020 was a virtual event this year due to COVID-19, but
MedPage Today s reporter, Ian Ingram, followed all the major developments from the conference. Ingram: One notable study at this year s ASH meeting found that older, high-risk patients with myelodysplastic syndromes, or MDS, lived significantly longer when they underwent allogeneic hematopoietic stem-cell transplantation.