Clinical Trials – New treatment for Hunter Syndrome.

Actigen is leading clinical trials for a pioneering new therapy, GNR-055, that could transform the St John’s treatment of MPS II (known as Hunter syndrome), a serious rare disease affecting one in 100,000–170,000 births
hunter syndrome

New treatment for Hunter Syndrome

MPS II is a rare, debilitating disease is caused by a deficiency of the key enzyme responsible for breaking down sulfated sugar molecules called glycosaminoglycans (GAGs) in cells throughout the body. If left untreated, this leads to a progressive deterioration of organ systems across the body, including the heart, lungs, brains, bone and cartilage. Brain deterioration leads to cognitive and behavioral issues such as disturbed sleep, hyperactivity, poor concentration, disruptive behavior and poor temper control.

The current treatment options available, such as enzyme replacement therapy, only offer a partial solution. While they can help improve the functioning of internal organs and improve patients’ quality of life, their inability to cross the blood-brain barrier means that GAGs continue to build up in the brain, resulting in ongoing neurological issues. (Boado et al 2014)

Existing treatments only offer a partial solution, improving the function of the patient’s internal organs, but not addressing the neurological issues.

GNR-005 uses an innovative combination of an enzyme and an antibody fragment to cross the brain-blood barrier to treat patients’ neurological issues. This treatment is a potential breakthrough and could offer life-changing improvements for patients with MPS II.

Located in St John’s Innovation Centre, Actigen’s clinical trial work forms part of a global strategic partnership with international pharmaceutical company Generium who manufactures one of the COVID-19 vaccines.

hunter syndrome

Managing director Michael Braunagel said: “MPS II is just one of thousands of rare diseases that have lacked clinical awareness, resources and treatment options. At Actigen, it is our mission to improve the lives of individuals with rare diseases, and we hope that GNR-055 will be the start of many more trials.”

Actigen’s clinical trial on GNR-055 will be undertaken as part of a global strategic partnership with Generium, a leading pharmaceutical company. Actigen’s team possesses specialist expertise in clinical trials and antibodies for rare diseases, and previously worked on development of a therapeutic antibody for global pharma giants Roche.

Michael Braunagel added: “We are establishing an exciting approach to clinical trials with GNR-055 and are working with our partner to bring products to global markets.

“MPS II is very debilitating condition for those affected and we are hopeful that the trial will establish the treatment’s efficacy.

“GNR-055 is the first therapeutic in our innovative development pipeline and we are thrilled to be working with Generium, who is a major player in the industry.”


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