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First gene therapy for hereditary blindness approved in the United States

First gene therapy for hereditary blindness approved in the United States


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FDA speaks of a milestone in gene therapy

The U.S. Food and Drug Administration (FDA) recently issued a press release announcing the approval of Luxturna gene therapy for hereditary eye disorders. The therapy is intended to treat children and adults who suffer from an inherited loss of vision caused by a gene mutation, which can result in blindness.

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

A turning point for new forms of therapy

Gottlieb described the approval of gene therapy as a turning point in relation to new types of therapy. "At the FDA, we focus on creating the right policy framework to benefit from this scientific opening," said Gottlieb in a FDA press release. Next year, the FDA will issue a series of disease-specific guidance documents for the development of gene therapy products to create modern and more efficient parameters for the evaluation and review of gene therapies.

How does the new therapy work?

Gene therapy Luxturna can alleviate some form of blindness caused by a defect in the RPE65 gene. In the United States, according to the FDA, up to 2,000 people have a mutation in both copies of the RPE65 gene. This mutation leads to a gradual loss of vision. In the newly approved gene therapy Luxturna, the non-mutated RPE65 gene is inserted directly into the retinal cells. The costly therapy has already been tested on more than 70 patients. 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 worldwide recognized specialist in the diagnosis of patients with RPE65 mutations. She welcomed the approval of gene therapy in the United States as a "big step". A corresponding application for Europe is currently pending with the responsible drug agency EMA for decision. According to Lorenz, the therapy cannot revive already dead sensory cells, but it can slow the further progression of the disease.

Spread of RPE65 disease in Germany

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

Author and source information



Video: Luxturna: Gene Therapy for Biallelic RPE65 Mutation-Associated Retinal Dystrophy (June 2022).