07:52 AM EDT, 09/05/2025 (MT Newswires) -- uniQure said Friday that initial safety and efficacy results from the first cohort of its early-to-mid stage trial of AMT-191, a gene therapy for Fabry disease, showed sustained increases in enzyme activity.

The drugmaker said the data showed that all four patients were able to discontinue enzyme replacement therapy while maintaining stable disease markers for up to 45 weeks post-treatment.

The company said the therapy also demonstrated a manageable safety profile.

In the higher-dose group, two patients experienced serious adverse events, including some possibly related to the treatment, the company said.

It added that a case of elevated liver enzymes was resolved with steroids and did not affect treatment expression.

No serious adverse events were reported in the lower-dose group, which includes three patients with limited follow-up so far, the company said.

Additional data from the dose-finding study is expected in the first half of 2026.