Voretigene neparvovec-rzyl

Voretigene neparvovec-rzyl is a gene therapy product used for the treatment of Leber's congenital amaurosis, a rare form of inherited blindness. It is the first in vivo gene therapy approved by the Food and Drug Administration (FDA) in the United States.

History

Voretigene neparvovec-rzyl was developed by Spark Therapeutics, a Philadelphia-based biotechnology company. The therapy was approved by the FDA in December 2017, making it the first directly administered gene therapy approved in the United States that targets a disease caused by mutations in a specific gene.

Mechanism of Action

Voretigene neparvovec-rzyl works by delivering a normal copy of the gene RPE65 directly into retinal cells. These cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient's vision function.

Clinical Trials

The approval of voretigene neparvovec-rzyl was based on a phase 3 trial involving patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The trial demonstrated the effectiveness of the therapy in improving the functional vision of patients.

Side Effects

Common side effects of voretigene neparvovec-rzyl include eye redness, cataract, increased intraocular pressure and retinal tear.

Future Developments

Research is ongoing to determine the long-term effects of voretigene neparvovec-rzyl and to expand its use to other forms of inherited retinal diseases.

See Also

• Gene therapy
• Leber's congenital amaurosis
• Food and Drug Administration
• Spark Therapeutics