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Breaking News - UK Gauchers Association Granted a Positive Opinion For Orphan Designation For Gene Therapy For Gaucher Disease

27 February 2015

The UK Gauchers Association is delighted to announce that on 12 February 2015 the Committee for Orphan Medicinal Products (COMP) at the European Medicine Agency (EMA) granted a positive opinion for orphan designation (EMA/OD/303/14) to the Association for adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene (Gene Therapy) as a new potential treatment for Gaucher disease.

Tanya Collin-Histed, Chief Executive of the Gauchers Association, said "Whilst existing enzyme replacement and substrate reduction therapies are effective in treating many areas of Gaucher Disease there remain significant unmet needs and challenges. No treatment is available to treat the neurological aspects of the disease which can range from eye movement and auditory processing to myoclonic seizure and premature death and gene therapy could potentially address this by providing a cure for Gaucher disease. Although this is a first step in the long and complex process of bringing a treatment to market, we are extremely excited to receive this opinion, and to initiate the procedures that allows for communicating directly with the regulators.”