Spanish clinical-stage genetic medicines company SpliceBio announced on Thursday that it has dosed the first patient in its Phase 1/2 ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease, an inherited retinal disorder caused by mutations in the ABCA4 gene.

SB-007 is an investigational Protein Splicing dual AAV gene therapy in development for the treatment of Stargardt disease. It is designed to restore expression of the native full-length ABCA4 protein in the retina. SB-007 has been granted Orphan Drug Designation from regulators in the United States and in Europe. In December 2024, SB-007 received US Food and Drug Administration (FDA) IND clearance, marking the first-ever clearance for a dual AAV gene therapy in Stargardt disease.

Miquel Vila-Perello, PhD., SpliceBio CEO and co-founder, said: "Treating the first patient in the ASTRA study with SB-007 is an important milestone for SpliceBio and the Stargardt disease patient community, who are in desperate need for a therapy. I am immensely proud of our outstanding team at SpliceBio whose depth of experience in leading numerous ophthalmology gene therapy trials in the last 15 years is unparalleled."

Paul Yang, M.D., Ph.D., chief of the Paul H. Casey Ophthalmic Genetics Division at Oregon Health & Science University Casey Eye Institute and principal investigator for the Phase 1/2 ASTRA Study, added: "Stargardt disease is a devastating inherited retinal disorder with no approved treatments available. The initiation of this study with the dual AAV vector gene therapy, SB-007, represents a critical advancement in finding a potential treatment option for patients with this disease."