‘s trial of its gene therapy for hemophilia in December when one of the 54 treated patients developed liver cancer. Over the weekend, the Dutch company announced that independent investigators had concluded that the cancer wasn’t caused by the gene therapy, which uses engineered viruses to add healthy genes to a patient’s DNA. The finding should alleviate worries that such gene insertions could cause cancer.
uniQure stock (ticker: QURE) rose nearly 5% in Monday trading, to $30.67, despite a roughly 1% drop in the
Nasdaq Composite index.
The company shared the findings with the U.S. Food and Drug Administration and hopes the agency will allow uniQure to now file for approval of the treatment for hemophilia B. It has finished dosing all patients in the Phase 3 trial.
Three outside researchers and