SpliceBio's SB-007 Gets FDA Nod for Stargardt Treatment Breakthrough

SpliceBio, a genetic medicines company, has announced that it has received clearance from the U.S. Food & Drug Administration (FDA) to move forward with an investigational new drug (IND) application for its lead program SB-007, which marks the first-ever IND clearance for a Protein Splicing gene therapy. This novel therapy is designed to target Stargardt disease, a genetic condition caused by mutations in the ABCA4 gene. Stargardt disease is a leading form of inherited juvenile macular degeneration, affecting about 1 in 8,000 to 10,000 individuals, and has no approved treatments to date due to the large size of the ABCA4 gene. SB-007 employs a dual AAV gene therapy to deliver the full-length ABCA4 gene, aiming to restore expression of the native ABCA4 protein in the retina. The FDA's clearance represents a major milestone as SpliceBio prepares to launch a Phase 1/2 clinical study (ASTRA) in early 2025. The study will assess the safety and efficacy of SB-007 and is informed by POLARIS, a natural history study launched by SpliceBio to understand disease progression in Stargardt disease. The company, supported by Ysios Capital, also plans to present at the J.P. Morgan Healthcare Conference in January 2025.