Stargardt disease is a rare disease which results in progressive vision loss. It is also referred to as juvenile macular degeneration. Most often, the disease presents during childhood. The most common cause of the condition is a mutation in the ABCA4 gene. Vitamin A byproducts buildup inside the photoreceptors in the eye because individuals with Stargardt are missing the protein responsible for clearing these away. This protein is made by a healthy ABCA4 gene. Without a healthy ABCA4 gene, the rods (which let you see in dim lighting) and the cones (which help you see color and fine detail) within the photoreceptors of the eye die.
The primary symptom of this condition is vision loss however, other symptoms include light sensitivity, color blindness, and seeing dark or hazy spots. The timeline of symptoms varies highly from person to person. In general, those who experience an earlier onset face a more rapid progression of symptoms.
There are currently no treatments for those living with Stargardt disease. Doctors simply recommend that patients protect their vision as much as possible by wearing hats or sunglasses when they are in bright lighting.
But there’s new exciting news for this patient community. An Investigational New Drug application has just been approved for a new potential oral therapy for this condition. It’s called LBS-008.
LBS-008 was developed by Belite Bio for both Stargardt disease and Age-related Macular Degeneration (AMD). Thanks to this new approval, Belite plans to begin initiation of a Phase 1 clinical trial. It will be a single ascending dose investigation of LBS-008.
LBS-008 works to prevent the buildup of vitamin A byproducts in the eye. The therapy has already received Orphan Drug Designation by both the FDA and the EMA as well as Rare Pediatric Disease Designation by the FDA.
Stay tuned to hear more about this upcoming clinical trial!
You can read more about this potential therapy and Belite Bio’s work in Stargardt disease here.