SpliceBio Announces First Patient Dosed in Phase 1/2 ASTRA Study of SB-007, a Dual-AAV Gene Therapy for Stargardt Disease
- SB-007 addresses the root cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations
- SB-007 is the first dual AAV gene therapy in clinical development for Stargardt disease
BARCELONA, SPAIN, 13 March 2025 – SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced that it has dosed the first patient in the Phase 1/2 ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease. Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene. It is the most common form of juvenile macular degeneration, leading to progressive central vision loss and ultimately resulting in blindness, with no approved treatments available.
