SB-007

**SB-007** is an investigational dual adeno-associated virus gene therapy developed by SpliceBio for autosomal-recessive Stargardt disease type 1 caused by biallelic pathogenic variants in the **ABCA4** gene. It delivers the large ABCA4 coding sequence in two AAV vectors and uses protein splicing to reconstitute expression of native full-length ABCA4 protein in the retina. SB-007 is administered as a single subretinal injection and is being evaluated in the Phase 1/2 ASTRA study. ([splice.bio](https://splice.bio/splicebio-announces-first-patient-dosed-in-phase-1-2-astra-study-of-sb-007-a-dual-aav-gene-therapy-for-stargardt-disease/))

Other names

AAV8-hABCA4AAV-8-hABCA4AAV 8-hABCA4Splicebio Stargardt gene therapy