Gene Therapy Treatment for Stargardt Disease given the go-ahead


Currently, there are no treatments available for Stargardt disease and it can often lead to the sufferer’s vision becoming very unclear and can even result in blank patches. However, a leading gene therapy company Oxford BioMedica have been given the go-ahead with their gene therapy for the disease - StarGen. They were given what’s called “orphan designation” from the European Medicines Agency (EMEA) which will basically give the company reduced regulatory fees and marketing exceptionality. The EMEA will only give this orphan designation to products that they think have the potential to provide an advantage over any treatments already in place for the condition.

Stargardt’s disease is a rare hereditary disorder that affects the macula area of the retina, as cells stop working causing the vision problems. This is thought to be caused by abnormalities of a gene called the ABCA4 gene and this company believe that they can correct this abnormal gene by replacing it with a correct one through gene therapy. The Chief Executive Officer of Oxford BioMedica, John Dawson, suggests that gene correction is the only means of solving the root cause of the disorder. Their technology that has already been evolved within the company (LentiVector®) can be used to place the genes in the necessary cells and can even place them in non-diving cells.

This news offers promise to thousands of people who suffer from the disease which can often be debilitating and hamper the sufferer’s life. With the orphan designation comes significant value to the development and regulation of the treatment and offers many commercial benefits too. It is hoped that the clinical development for this gene therapy is to start in the New Year, alongside Sanofi-Aventis (one of the worlds leading pharmaceutical companies). The treatment has also received previous funding and continued backing from the American charity Foundation Fighting Blindness. With the numerous backing and voices of confidence supporting this campaign it suggests that StarGen is something to look out for in the not so distant future with regard to Stargardt’s disease.

16th March 2010

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