Karen Wai, MD
Byers Eye Institute at Stanford University
Palo Alto, CA
Monday afternoon of ASRS was the Hereditary Retinal Disease & Genetics symposium, moderated by Dr. Diana Do and Dr. David Saperstein. The talks included two late-breaker presentations and a talk on complications of transvitreal subretinal injections.
The first late-breaking abstract was presented by Dr. Allen Ho, who discussed the 6-month outcomes from the phase II STARLIGHT trial. He discussed MCO-010 optogenetic therapy for patients with Stargardt’s disease. MCO-010 aims to re-sensitize the retina by using an optogenetic molecule to target bipolar cells. The trial included 6 patients over a median of 28 weeks. The study found no serious adverse events, and no systemic events related to MCO-010. Patients with predominantly macular disease showed improvements in visual acuity. While those who had widespread disease had no improvement in acuity. Dr. Ho also shared an inspiring patient video describing her improvement after her treatment with optogenetic therapy.
The second late-breaking abstract was presented by Dr. Quan Dong Nguyen. He discussed the 18-month results of Tinlarebant (LBS-008) for adolescent patients with Stargardt’s disease. Tinlarebant is an oral specific non-retinoid antagonist of retinol binding protein 4 (RBP4), which is thought to slow disease progression in Stargardt disease.The phase 1b/2 study found that Tinlarebant was well-tolerated without any reported moderate or severe drug-related adverse events. We look forward to the results of the phase 3 study of Tinlarebant in adolescent subjects with STGD1 (the DRAGON study), which has recently finished recruiting.
Dr. Chloe T Koo discussed frequency of complications surrounding transvitreal subretinal injections for gene therapy vectors in inherited retinal disease (IRD). A retrospective review was performed on 70 eyes of 46 patients who had transvitreal subretinal injections for IRDs including Leber congenital amaurosis, X-linked retinitis pigmentosa, choroideremia, and achromatopsia. She described that complications are relatively common, and were grouped as surgical procedure-related (which included retinal tears, foveal schisis, RPE atrophy from cannula touchdown, choroidal neovascularization, and RPE stretching), vector-related (chorioretinal atrophy), and steroid-related complications. She described methods to minimize and prevent possible complications related to these transvitreal subretinal injections, and that the technique will continue to be modified to reduce complications. Ultimately, careful selection of patients may determine final visual outcomes.
