A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene
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Summary
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1). This is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.
Description
Inherited retinal dystrophies (IRDs) are a group of genetically and clinically heterogeneous disorders caused by progressive degeneration of photoreceptor (PR) rods, cones, or both. The majority of IRDs are characterized by retinal degeneration, which can lead to significant vision impairment and blindness and are thus serious and debilitating conditions. Most IRDs have no treatment options. Stargardt disease (STGD1) is an autosomal recessive IRD that generally manifests during childhood or early adulthood. It has an estimated prevalence of 1 in 8,000 to 10,000 patients. STGD1 is due to mutat…