Safety Seen in AMT-130 Gene Therapy Trial, Six More Patients to Enroll

Safety Seen in AMT-130 Gene Therapy Trial, Six More Patients to Enroll

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Safety Seen in AMT-130 Gene Therapy Trial, Six More Patients to Enroll

No significant safety concerns were found in the first four patients participating in a Phase 1/2 trial assessing the safety and efficacy of uniQure’s AMT-130, an experimental gene therapy for Huntington’s disease. This finding — the outcome of a second meeting with the independent Data Safety Monitoring Board (DSMB) overseeing the proof-of-concept trial (NCT04120493) — opens the way for enrolling and treating another six people. uniQure expects to finish recruiting a full first group of…

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