uniQure (QURE.US) soared due to the gene therapy AMT-130, Goldman Sachs is bullish: Peak sales are expected to reach $2.5 billion.

Wisdom Financial APP learned that Goldman Sachs' latest in-depth analysis report focuses on uniQure NV (QURE.US) gene therapy AMT-130 breakthrough progress and commercial prospects in the treatment of Huntington's disease. According to disclosures, the therapy has shown significant efficacy in two dose groups in clinical trials - after 36 months of follow-up for 17 patients in the high-dose group, the composite Huntington's disease assessment scale showed a 75% slowing of disease progression compared to natural history, a 60% slowing of total functional capacity score changes, and an average 8.2% decrease in cerebrospinal fluid neurofilament light chain protein levels; although direct comparative data was not provided for the 12 patients in the low-dose group, the overall trend was consistent with the high-dose group. Biomarkers and multiple secondary endpoints all support the neuroprotective effect of AMT-130. Recently, the stock has surged due to this therapy news.