Uniqure NV
F:UQ1
Uniqure NV
uniQure NV engages in the discovery, development, and commercialization of innovative gene therapies. The company is headquartered in Amsterdam, Noord-Holland and currently employs 463 full-time employees. The company went IPO on 2014-02-05. The company seeks to develop one-time administered treatments with potentially curative results for patients suffering from genetic and other devastating diseases. The company develops, both internally and through partnerships, a pipeline of gene therapies. The company produces adeno-associated virus based, or AAV-based, gene therapies in its own facilities with a proprietary, commercial-scale, current good manufacturing practices, compliant, manufacturing process. AMT-061, the Company’s lead product candidate for patients with hemophilia B, is going through a dosing phase of a pivotal study. AMT-130, the product candidate for patients with Huntington’s disease is in Phase I/II clinical study.
uniQure NV engages in the discovery, development, and commercialization of innovative gene therapies. The company is headquartered in Amsterdam, Noord-Holland and currently employs 463 full-time employees. The company went IPO on 2014-02-05. The company seeks to develop one-time administered treatments with potentially curative results for patients suffering from genetic and other devastating diseases. The company develops, both internally and through partnerships, a pipeline of gene therapies. The company produces adeno-associated virus based, or AAV-based, gene therapies in its own facilities with a proprietary, commercial-scale, current good manufacturing practices, compliant, manufacturing process. AMT-061, the Company’s lead product candidate for patients with hemophilia B, is going through a dosing phase of a pivotal study. AMT-130, the product candidate for patients with Huntington’s disease is in Phase I/II clinical study.
AMT-130 Results: uniQure reported positive top-line Phase I/II data for AMT-130 in Huntington's disease, showing a significant slowing of disease progression.
FDA Setback: The FDA now questions whether current data are sufficient for a BLA filing, introducing uncertainty and delaying the timeline.
Robust Data: Management emphasized the strength and consistency of AMT-130’s results across multiple analyses.
Financial Position: Cash and investments were $694.2 million at quarter-end, expected to fund operations into 2029.
Pipeline Updates: Encouraging early results in Fabry disease and mesial temporal lobe epilepsy; ALS program enrollment paused due to a safety event.
Outlook: uniQure is urgently engaging with the FDA and planning to approach EU/UK regulators for AMT-130.
