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uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma (HCC) in Hemophilia B Gene Therapy Program
~ Independent investigation shows that etranacogene dezaparvovec is highly unlikely to be the causeof HCC in HOPE-B pivotal trial ~ ~ Data from investigation
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[{"type":"text","content":"~ Independent investigation shows that etranacogene dezaparvovec is highly unlikely to be the causeof HCC in HOPE-B pivotal trial ~ ~ Data from investigation submitted to U.S Food and Drug Administration (FDA) on March 26, 2021 ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, March 29, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today the results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec. The results of the investigation found that it is highly unlikely the HCC was caused by etranacogene dezaparvovec. Multiple analyses conducted by an independent laboratory and reviewed by leading external experts in the field show that AAV vector integration in the patient’s tissue sample was extremely rare and accounted for 0.027% of the cells in the sample. The integration events were distributed randomly across the genome, and there was no evidence of clonal expansion or any dominant integration event. Additionally, whole genome sequencing of the tumor confirmed that the patient had several genetic mutations that are characteristic of HCC and are independent of vector integration. Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC. “This investigation has employed several complementary genomic approaches to evaluate the involvement of the AAV vector in the development of the liver cell cancer in this patient,” stated David Lillicrap, M.D., FRCPC, Professor of the Department of Pathology and Molecular Medicine, Queen’s University, Kingston, Canada. “The investigations that have been performed have shown no evidence to suggest that the AAV vector delivered in the HOPE-B study has played a pathogenic role in the hepatocellular cancer that has now been diagnosed in the patient.” “The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC,” stated Ricardo Dolmetsch, president of research and development at uniQure. “Moreover, whole genome sequenc...