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UniQure (QURE) Reports Positive Phase I/IIa AMT-191 Data for Fabry Disease, Shows Sustained Enzyme Activity

UniQure (NASDAQ:QURE) is one of the most promising low-cost stocks to buy now. On February 6, UniQure reported positive updated data from its Phase I/IIa trial of AMT-191, which is a gene therapy for Fabry disease. All 11 patients across three different dose levels showed increased enzyme activity, with the longest-treated patient maintaining these high levels for over a year. Notably, the enzyme levels were significantly above normal ranges, showing a clear relationship between the dose received and the amount of enzyme produced.

The treatment allowed 6 out of the 11 patients to stop their regular enzyme replacement therapy/ERT after meeting specific clinical criteria. Even after stopping ERT, patients maintained stable levels of lyso-Gb3, the toxic substance that typically builds up in Fabry disease. This suggests that the one-time gene therapy could potentially replace the need for lifelong, bi-weekly infusions for some patients.

While the therapy shows promise, UniQure has paused new dosing in the mid- and high-dose groups to evaluate some safety findings. Two patients in the middle dose group experienced high liver enzyme levels that were classified as dose-limiting toxicities, though both responded to steroid treatment. Other previously reported serious events in the high-dose group included chest pain and a stroke, though some were determined to be unrelated to the study drug.

UniQure (NASDAQ:QURE) develops treatments for patients suffering from rare and other devastating diseases in the US.

While we acknowledge the potential of QURE to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than QURE and that has 100x upside potential, check out our report about this cheapest AI stock.

READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now.

Disclosure: None. This article is originally published at Insider Monkey.

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