FDA approves unconventional gene therapy



The approval of Luxturna by FDA marks yet another case of success achieved by gene therapies, while it also proves that such treatments may constitute an important milestone not only with regard to curing eye diseases but also types of cancer and many incurable diseases. There were three gene therapies targeting serious and rare illnesses approved last year alone.

The new product is intended for the treatment of patients having been diagnosed with a mutation of the RPE65 gene which leads to retinal dystrophy. The disease causes visual dysfunction and usually leads to a complete loss of sight. Congenital dystrophies represent a broad category of genetic disorders of the retina. It is estimated that they depend on approx. 220 various genes. Problems caused by the defective RPE65 gene are related to approx. 1,000-2,000 patients in the territory of the U.S. The disabled gene impairs the production of the protein that catalyzes chemical reactions and is responsible for proper vision. A worsened quality of sight is usually present as early as in one's childhood, being subject to continuous progress.

The working principle of Luxturna is based on the provision of a fine version of the gene directly to one's retinal cells which, after having been subject to such manipulation, begin producing the protein that converts light into an electrical signal. Due to this, the patient's vision becomes restored. Such substitution of genetic material is possible by using adenoviruses that carry and deliver the gene prepared with the use of DNA recombinant technologies.

The testing of a new product was conducted on a group of 41 patients aged 4-44 who have been diagnosed with the mutation of RPE65 gene. Their condition was characterised by a significant improvement in the quality of sight, particularly at low-light levels. Adverse reactions reported with regard to this therapy included: eye redness, cataract, increased intraocular pressure and teary eyes. While the preparation may only be applied to patients with live retinal cells, it still provides a chance of vision restoration to thousands of patients around the world.


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