The company's vision is to build an industry-leading, fully-integrated and global company that leverages its validated technology and manufacturing platform to deliver transformative gene therapy products to patients with serious unmet medical needs.

uniQure is leveraging its modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases.

The company's pipeline of adeno-associated virus (AAV)-based gene therapies is developed both internally and through multiple collaborations using its innovative, modular technology platform. Through collaboration with Bristol-Myers Squibb, for the development of gene therapies for cardiovascular diseases, uniQure has made steps towards developing gene therapies targeting chronic and degenerative diseases that affect larger populations.

In January 2023, uniQure entered into a global licensing agreement with Apic-Bio for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders. The US Food and Drug Administration has cleared the investigational new drug (IND) application for APB-102 and has granted Orphan Drug and Fast Track designations for the product.