gene therapy

Harriet Farmer's picture

First Gene Therapy Wins Backing in Europe – the Start of Things to Come

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At €1 million a pop, expectations are high.  [[wysiwyg_imageupload:134:]]
But European regulators have given the initial thumbs up to Glybera (alipogene tiparvovec), the western world’s first gene therapy, paving the way for approval by the European Commission.

The treatment is for a rare type of pancreatic disorder in which patients have damaged copies of a gene which is essential for breaking down the fat content in foods.  Affecting one person in a million, the disease causes a build up of lipids in the blood stream, leading to abdominal pain and potentially fatal inflammation of the pancreas.

The decision marks an important advance for gene therapy, a field which has been hotly debated for many years due to shortcomings of previous investigational therapies.  Approval for Glybera, which was previously rejected on three occasions, will make it the first gene therapy to be available outside a clinical trial in Europe or the US.  China approved a gene therapy for head and neck cancer in 2004. Read full post »