Over the past 30 years, gene therapy has been developed as a powerful treatment modality for IRDs, with over 100 clinical trials having been conducted in patients.  

Luxturna, an adeno-associated viral vector carrying the RPE65 gene for the treatment of type 2 Leber congenital amaurosis, has been approved by the US FDA as the first gene therapy product for IRDs.  

While promising results have also been obtained in clinical studies for  choroideremia,  X-linked retinitis pigmentosa, and Leber’s hereditary optic neuropathy, no significant improvement has been reported in clinical studies of X-linked retinoschisis and Stargardt disease.  

We are developing gene therapies for rare ocular diseases with significant unmet medical need.