Ocugen Launches Phase 2/3 Clinical Trial for Stargardt Disease Gene-Modifier Therapy
Research News
The therapy delivers copies of the RORA gene to address multiple disease pathways.
The biotechnology company Ocugen has dosed the first patient in its Phase 2/3 GARDian3 clinical trial for OCU410ST, an emerging gene-modifier therapy for people with Stargardt disease caused by mutations in the gene ABCA4.
According to Ocugen, OCU410ST is designed to deliver copies of the RORA gene to retinal cells to reduce the formation of lipofuscin, the harmful deposits in the retina which are the hallmark of Stargardt disease. The company believes the treatment can also reduce oxidative stress, the formation of complement (harmful immune system proteins), and inflammation.
OCU410ST use a human-engineered adeno-associated virus (AAV) to deliver copies of the RORA gene to retinal cells. The therapy is administered using a one-time, subretinal injection.
The Phase 2/3 clinical trial will enroll 51 participants with 34 receiving OCU410ST in their worse-seeing eye and 17 participants assigned to the control (untreated) group.
The company reported encouraging safety and efficacy results for six patients in its Phase 1/2 clinical trial for OCU410ST.
