A uniQure gene therapy for Huntington’s disease has interim clinical data showing an 80% slowing of disease progression. An accelerated approval pathway is one of the topics the company wants to discuss with the FDA.
By selling partial royalties to approved hemophilia gene therapy Hemgenix, uniQure gains immediate cash to support its pipeline, including a Huntington’s disease gene therapy already in the clinic . The royalty deal extends uniQure’s cash runway into 2026.
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A uniQure gene therapy for hemophilia B in on track for an FDA submission in the first quarter of 2022. Meanwhile, uniQure added to its pipeline with the acquisition of preclinical epilepsy gene therapy biotech Corlieve Therapeutics for €46.3 million upfront.
A uniQure gene therapy for hemophilia B that has been under a clinical hold for four months can now resume testing. The hold was lifted after uniQure answered the FDA's questions about whether the gene therapy caused the liver cancer diagnosed in one patient who received the therapy.
UniQure is making a case for lifting a clinical hold on its hemophilia B gene therapy, reporting Monday results of an independent investigation that appear to clear the experimental treatment as the cause of a patient’s cancer.
The therapy, etranacogene dezaparvovec, is currently in Phase III clinical development for hemophilia B. Shares of uniQure were down nearly 20%, but an investment bank analyst wrote that the financial terms were favorable for the company.
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