uniQure has built an industry-leading gene therapy platform focused on liver-directed and central nervous system (CNS) disorders.
Gene therapy offers the prospect of long-term treatments and potential cures to patients with genetic or acquired diseases. Research studies show our AAV platform can restore the expression of a missing protein or block the expression of a gene, after a single administration.
Genes provide the blueprint used by cells to make proteins. A defect or mutation in a gene can prevent the production of a protein, change its function, or make a protein toxic.
For example, when the gene associated with the production of factor IX, a protein required for blood clotting, is missing or mutated in people with hemophilia B, they bleed even after minor trauma or surgery. Introducing a copy of the missing gene into the liver restores the levels of factor IX in the blood and reduces bleeding. Similarly, mutations in the huntingtin gene result in the production of a mutant protein that causes degeneration of neurons in the brain in patients with Huntington’s disease. Introducing a gene therapy into the brain that may prevent the expression of the huntingtin gene. A clinical program is ongoing to assess the potential to prevent brain degeneration in these patients.
uniQure develops its gene therapies using an innovative and modular technology platform, that leverages our proprietary manufacturing process.
The platform is modular in that it allows us to develop, manufacture and clinically test gene therapies for multiple diseases using the same set of components, including capsids, promoters, and manufacturing methods. In many cases, the disease-specific gene cassette is the only component that needs to be changed to target a new disease. We believe we will be able to reduce the time, cost, and risk of developing treatments while offering medicines that are effective and convenient alternatives to the current standard of care.