uniQure N.V.

uniQure N.V. is a biotechnology company focused on developing gene therapies for patients with rare and devastating diseases. Its only marketed product is HEMGENIX, a gene therapy for hemophilia B that enables patients to produce factor IX, reducing bleeding risk. The company's pipeline includes several investigational candidates: AMT-130 for Huntington's disease (Phase I/II), AMT-260 for mesial temporal lobe epilepsy (Phase I/IIa), AMT-162 for SOD1-ALS (Phase I/IIa), and AMT-191 for Fabry disease (Phase I/IIa). uniQure also has licensing and supply agreements with partners like Apic Bio and CLS Bhering.

uniQure is listed on the Nasdaq Global Select Market (NasdaqGS) under the ticker symbol QURE. Its shares trade in US dollars (USD), as the company is classified in the US region for listing purposes, despite being headquartered in the Netherlands.

uniQure operates in the Healthcare sector, specifically within the Biotechnology industry. This means the company is engaged in researching and developing biological therapies—in this case, gene therapies—to treat medical conditions, primarily rare and serious diseases.

uniQure is headquartered in Amsterdam, the Netherlands, and was founded in 1998. While based in Europe, the company primarily serves the United States market, as indicated by its description summary noting it develops treatments for patients in the US. Its commercial product HEMGENIX and clinical trials are focused on the US healthcare system.

uniQure's pipeline includes several gene therapy candidates in early-to-mid-stage clinical trials. AMT-130 is in Phase I/II for Huntington's disease, a neurodegenerative disorder. AMT-260 is in Phase I/IIa for mesial temporal lobe epilepsy. AMT-162 targets SOD1-ALS, a form of amyotrophic lateral sclerosis, also in Phase I/IIa. AMT-191 is an investigational therapy for Fabry disease, currently in Phase I/IIa. Additionally, uniQure has a licensing agreement with Apic Bio for an intrathecally administered gene therapy for SOD1-ALS and a supply agreement with CLS Bhering.