Gene Therapy for Retinitis Pigmentosa

Tested in Humans for the first time.

French Biotech Horama has treated the first patients with a gene therapy for retinitis pigmentosa that could prevent the progressive loss of vision caused by this genetic condition

A Phase 1/11 trial with Horama’s gene therapy for retinitis pigmentosa is running at the Nantes University Hospital. The therapy is aimed at fixing genetic defects in a gene called PDE6b, which causes retinal cells to progressively die as patients age, worsening their vision over time.

The trial will recruit a total of 12 patients, which will receive an injection of the gene therapy in one of their eyes. The first six patients will receive one of two doses of the gene therapy, and once the optimal dose is selected the results will be confirmed in the remaining patients. Their progress will be followed over time and results will be collected a year after the patient receives the one-off treatment.