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...

07:58 AM EDT, 09/05/2025 (MT Newswires) -- Regenxbio ( RGNX ) said Friday that longer-term data from a phase I/II/III trial of clemidsogene lanparvovec, or RGX-121, further showed the ability of one-time treatment to improve outcomes for patients with Mucopolysaccharidosis Type II, or Hunter syndrome. The company said it has submitted the 12-month data to the US Food and Drug...

07:52 AM EDT, 09/05/2025 (MT Newswires) -- CG Oncology ( CGON ) said Friday that updated data from a phase 3 trial of its investigational drug cretostimogene as a monotherapy for patients with high-risk non-muscle invasive bladder cancer showed a robust 41.8% complete response rate at 24 months. The latest data came from a group of patients with high-risk non-muscle...

07:55 AM EDT, 09/05/2025 (MT Newswires) -- Braze (BRZE) shares were up 21% in recent Friday premarket activity, a day after the company reported higher fiscal Q2 adjusted earnings and revenue in addition to raising its fiscal 2026 guidance. Samsara (IOT) stock was up 12% after the company overnight posted higher fiscal Q2 adjusted net income and revenue in addition...