Hemophilia B Gene Therapy AMT-061 Safe, Effective, Trial Data Show

Hemophilia B Gene Therapy AMT-061 Safe, Effective, Trial Data Show

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Hemophilia B Gene Therapy AMT-061 Safe, Effective, Trial Data Show

AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with pre-existing antibodies against the treatment’s viral carrier, according to new trial data. Data from the Phase 3 HOPE-B trial suggest that nearly all hemophilia B patients, regardless of pre-existing viral inhibitors, may benefit from AMT-061. “Patients in the trial who may not have been eligible for other gene therapies because of pre-existing neutralizing antibodies have achieved similar results…

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