First gene therapy against hereditary blindness in the US approved

FDA speaks of a milestone in gene therapy

U.S. The Food and Drug Administration (FDA) recently announced in a press release the approval of Luxturna gene therapy for hereditary eye disease. The therapy will treat children and adults who suffer from a hereditary gene mutation-induced vision loss that may result in blindness.

"This milestone demonstrates the potential of the breakthrough treatment approach to treat a wide range of challenging diseases," said FDA Commissioner Scott Gottlieb about the approval of gene therapy. "The culmination of decades of research has led to three gene therapy approvals for patients with severe and rare diseases this year. I believe that gene therapy will be a major pillar in the treatment and perhaps cure of many of our most devastating and persistent diseases, "said Gottlieb.

A turning point for novel types of teapots

Gottlieb described the approval of gene therapy as a turning point in terms of novel forms of therapy. "At the FDA, we are focused on creating the right policy framework to benefit from this scientific opening," said Gottlieb in a press release from the FDA. Next year, the FDA will issue a set of disease-specific guideline documents for the development of gene therapy products to create more modern and more efficient parameters for evaluating and reviewing gene therapies.

How does the new therapy work??

Luxturna gene therapy can alleviate some form of blindness caused by a defect in the RPE65 gene. In the US, up to 2,000 people have a mutation in both copies of the RPE65 gene, according to the FDA. This mutation leads to a gradual loss of vision. In the newly approved Luxturna gene therapy, the unmutated RPE65 gene is directly incorporated into the retinal cells. The costly therapy has been tested on more than 70 patients. At the moment it is still unclear how long the positive effect will last.

Will gene therapy soon be approved in Europe??

The Director of the University Hospital Giessen Professor Birgit Lorenz is a globally recognized specialist in the diagnosis of patients with RPE65 mutations. She welcomed the approval of gene therapy in the US as a "big step". A request for Europe is currently being considered by the competent drug agency EMA. According to Lorenz, although the therapy can not revive already dead sensory cells, but slow down the further progression of the disease.

Distribution of RPE65 disease in Germany

The complex light sensor of the human eye is prone to hereditary diseases. "More than 250 different genetic visual disorders are known," explains the President of the German Ophthalmological Society (DOG) Professor. med. Thomas Kohnen in a press release of the DOG. Fortunately, most of them are rare. According to DOG, there are approximately 150 to 200 patients with RPE65 disease in Germany. (Fp)