Our Clinical Trial Solutions
Our current trial is focused on GNR-055, a fusion protein as a potential therapy for mucopolysaccharidosis II
(MPS II, Hunter syndrome)
GNR-055
We are working in partnership with Generium to trial GNR-055, a recombinant fusion protein specifically designed for treating mucopolysaccharidosis II (MPS II, Hunter syndrome). Generium is one of the leading biologicals developers in Russia, with a portfolio of approved medicines, an exciting pipeline of new medicine candidates, and a strong focus on orphan medicines. Generium is the manufacturer of a COVID-19 vaccine
Current enzyme replacement therapy does not treat the progressive neurological impairment associated with MPS II because iduronate-2-sulfatase (IDS) is a large molecule that cannot cross the blood-brain barrier.
Scientists have designed GNR-055 as a fusion protein of IDS linked to an antibody fragment that binds specifically to the insulin receptor on blood capillaries. When the antibody fragment binds to these insulin receptors, it can transport IDS across capillary walls into cells, including across the blood-brain barrier.
The brain has a rich supply of insulin receptors, so GNR-055 is transported selectively across the blood-brain barrier when administered by IV infusion.
Watch our video to learn about GNR-055’s mode of action
Treatment
GNR-055 is administered intravenously. It has the potential to stabilise or slow the progression of MPS II disease. As well as its effects elsewhere in the body, GNR-055 crosses the blood-brain barrier and is expected to relieve signs and symptoms related to the accumulation of GAGs in the brain.
Our MPS II Clinical Trial
Our planned clinical trial will evaluate the efficacy, safety, and tolerability of GNR-055 in all MPS II patients, with a primary focus on those with the most severe (neuronopathic) disease.