Actigen moves closer to clinical trial for rare disease treatment  


Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA).   

The Cambridge-based biotech filed for a pre-IND advice meeting with the FDA to discuss the scientific, regulatory and safety issues related to developing GNR-055 – the company’s new rare disease treatment for mucopolysaccharidosis II, otherwise known as MPS II or Hunter syndrome.  

Following what Actigen calls a positive response to the meeting, the company is now submitting its IND application and will begin clinical trials for GNR-055 if it receives FDA approval. 

Hunter syndrome 

Hunter syndrome is a rare, inherited disorder in which the body is unable to break down large sugar molecules, the build-up of which can damage organs and tissues in the body. It occurs primarily in males and current treatment options such as enzyme replacement therapy, only offer a partial solution. While they can help improve the patient’s internal organ function, they cannot cross the blood-brain barrier, resulting in ongoing neurological issues.  


GNR-055 works through a combination of an Iduronate 2-Sulfatase (IDS) enzyme and antibody fragment to access the brain cells. The idea behind the treatment is that it breaks down the glycosaminoglycans (GAGs) that cause neurological manifestation in the brain. 

Official comments 

Actigen’s Managing Director, Michael Braunagel, said: “The positive response from the pre-IND meeting strengthens our case for clinical trials in the US and shows that our approach is valid. We are looking forward to continuing our discussions with the FDA on specific issues raised and are optimistic to be in a position to conduct our clinical trials in the near future. GNR-055 is a potentially game-changing treatment for MPS II patients, with the ability to address the serious neurological difficulties they experience.  

MPS II has serious, life-limiting effects on those affected, and we are hopeful that our forthcoming clinical trials will demonstrate the treatment’s ability to transform patients’ quality of life. We are confident that we will successfully progress to the IND stage based on our initial discussions.”  

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