Splice Bio
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.
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…
Inclusion Criteria: To be eligible for study participation, subjects must meet the following criteria: 1. Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same. 2. Are male or female adolescents and adults, aged as follows: 1. In Part A, subjects will be ≥18 to ≤65\* years (inclusive) 2. In Part B, subject age is planned as ≥12 to ≤65\* years (inclusive) \*Subjects aged \>65 years may be eligible in Parts A and B, following discussion with, and approval by, the Medical Monitor. 3. Are able to understand and co…
Subretinal Administration of SB-007