A breakthrough treatment for Huntington’s could move from trials to real-world use sooner than expected, and that shift could change how this company is viewed. The catch is that everything hinges on regulators, safety results, and the company’s ability to manufacture and deliver the therapy without setbacks.Read more
Catalysts About uniQure uniQure develops gene therapies targeting serious neurological and rare metabolic diseases. What are the underlying business or industry changes driving this perspective?Read more
Catalysts About uniQure uniQure develops gene therapies targeting severe neurological and metabolic rare diseases. What are the underlying business or industry changes driving this perspective?Read more
uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in phase I/II clinical study for the treatment of Huntington’s disease. The company also develops AMT-260, which is in phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
