uniQure N.V. (uniQure) is a gene therapy company. The Company is engaged in developing single treatments with curative results for patients suffering from genetic and other severe diseases. The Company is advancing a focused pipeline of gene therapies that have been developed both internally and through partnerships. The Company’s product candidates include AMT-060 for Hemophilia B, AMT-130 for Huntington's disease, S100A1 for congestive heart failure and Glybera for lipoprotein lipase deficiency (LPLD). uniQure has established clinical proof-of-concept in its lead indication, hemophilia B and has achieved pre-clinical, proof-of-concept in Huntington's disease. The Company, through its collaboration with Bristol Myers-Squibb (BMS), is focused on building a portfolio of gene therapies led by the S100A1 program for congestive heart failure.
AMT-060 for Hemophilia B
AMT-060 is a gene therapy for the treatment of Hemophilia B. Hemophilia B is an inherited disease in males characterized by insufficient blood clotting. AMT-060 consists of the adeno-associated virus (AAV) vector carrying a human Factor IX (FIX) gene cassette that the Company has exclusively licensed from St. Jude Children’s Research Hospital in Memphis, Tennessee. The Company produces this vector with its insect cell-based manufacturing process. It has an ongoing Phase I/II clinical trials.
AMT-130 for Huntington's Disease
AMT-130 is developed for the treatment of Huntington's disease (HD). HD is caused by an inherited defect in a single gene that codes for protein called Huntingtin. The Company’s product candidate AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA, which silences the Huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein.
S100A1 for Congestive Heart Faliure
The Company has entered into an agreement with BMS that provides exclusive access to the Company’s gene therapy technology platform for multiple targets in cardiovascular (and other) diseases (Collaboration and License Agreement). The collaboration includes its proprietary gene therapy program for congestive heart failure, which aims to restore the heart's ability to synthesize S100A1. . S100A1 is a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction. S100A1 deficient hearts show accelerated progression to severe heart failure and increase mortality after cardiac damage. The Company is leading the discovery, non-clinical, analytical and process development effort and is responsible for manufacturing of clinical and commercial supplies using our vector technologies and industrial, proprietary insect-cell based manufacturing platform, while BMS leads development and regulatory activities across all programs and is responsible for all research and development costs. BMS will be solely responsible for commercialization of all products from the collaboration.
Glybera for Lipoprotein Lipase Deficiency
Glybera is a gene therapy that is designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal. The product consists of an engineered copy of the human LPL gene packaged with a tissue-specific promoter in a non-replicating AAV1 vector, which has a particular affinity for muscle cells. In order to improve activity, the Company uses a naturally occurring variant of the LPL gene that has higher enzyme activity than the normal version of the gene that encodes the protein. It produces Glybera using its insect cell-based manufacturing process. In the European Union, the Company has been granted orphan drug exclusivity for Glybera for treatment of LPLD until October 2022. The first commercial patient in Europe was treated with Glybera in September 2015.
Other Early-Stage Research
The Company is also engaged in the research of several other gene therapy candidates targeting rare and orphan diseases. It focuses on genetic diseases affecting the liver, including hemophilia A, as well as various central nervous system (CNS) disorders.
uniQure seeks to develop next-generation vectors with increased potency to target liver indications in which high relative percentage increases in the secretion of a protein above the disease state would be required for therapeutic benefit. These next-generation vectors may be used in the development of a gene therapy for hemophilia A as well as other therapeutic indications. In January 2014, the Company has entered into a collaboration and license agreement with 4D Molecular Therapeutics, LLC for the discovery and optimization of next-generation AAV vectors targeting the liver and the brain.
The Company competes with AGTC, Abeona Therapeutics, Adverum Biotechnologies, Asklepios BioPharmaceutical, Audentes Therapeutics, Avalanche Biotech, AveXis, Bayer, BioMarin, Bioveratiy, bluebird bio, Dimension Therapeutics, Errant Gene Therapeutics, Expression Therapeutics, Freeline Therapeutics, Genethon, Genzyme, GlaxoSmithKline, Homology Medicines, Lysogene, Megenics, Milo Therapeutics, Nightstarx, Pfizer, REGENXBIO, Renova Therapeutics, Retrosense Therapeutics, Sangamo BioSciences, Shire, Solid Biosciences, Spark Therapeutics, Takara, Voyager, Amgen, Bayer, Biogen, BioMarin, Genzyme, Novartis, Novo Nordisk, Pfizer, and Shire.