Phase I/II Clinical Trial of AMT-130
uniQure has initiated the first gene therapy clinical trials in Huntington’s disease (HD). uniQure has begun dosing patients in a randomized, double-blinded, sham controlled Phase I/II clinical study to assess the safety and tolerability of a one-time treatment of AMT-130 in patients with HD in the US (ClinicalTrials.gov Identifier: NCT04120493).
Phase III HOPE‑B Pivotal Study of etranacogene dezaparvovec (AMT-061) in Hemophilia B
The Phase III HOPE‑B trial is the largest and first pivotal trial of a gene therapy for patients with hemophilia B. It is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult hemophilia B patients classified as severe or moderately severe (defined as less than or equal to 2% of normal FIX activity) and requiring prophylactic FIX replacement therapy were enrolled in a prospective, six-month observational period during which time they continued to use their current standard of care therapy to establish a baseline annualized bleeding rate. No prophylactic immunosuppression was provided to patients upon entering the study. After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5. Forty-three percent of patients in the study had pre-existing NAbs to AAV5 up to a maximum observed pre-dosing titer of over 3,200.