AMT-130 Gene Therapy Appears Safe; More Data Forthcoming

AMT-130 Gene Therapy Appears Safe; More Data Forthcoming

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AMT-130 Gene Therapy Appears Safe; More Data Forthcoming

No significant safety concerns were identified in the first eight Huntington’s disease patients treated with uniQure’s potential gene therapy AMT-130 in a U.S. Phase 1/2 clinical trial. Six of these patients received the low dose of the therapy — which is delivered directly into the brain — and two received the high dose. In parallel, six additional participants underwent a sham brain surgery, four in the low-dose group and two in the high-dose group. This brings…

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