The first patient has been dosed in the Phase 1/2 ASTRA study for Stargardt disease gene therapy SB-007.

SpliceBio from Barcelona, Spain, announced on March 13^th the start of the trial which will assess SB-007, a dual adeno-associated viral (AAV) vector. The study is being conducted at the Casey Eye Institute in Oregon, USA. Paul Yang, MD, is principal investigator of the trial.

Stargardt Disease is the most common form of juvenile macular degeneration and is most often caused by mutations in the ABCA4 gene on Chromosome 1. This is a particularly large gene and over 1000 disease causing mutations have been identified. Due to the large size of the gene, traditional gene therapy methods using a single AAV virus was not possible. The ASTRA study uses a dual AAV vector protein splicing therapy, which is designed to restore expression of the native full-length ABCA4 protein in the retina.

“SB-007, represents a critical advancement in finding a potential treatment option for patients with this disease.” said Professor Yang.

For the full report : https://europe.ophthalmologytimes.com/view/first-patient-dosed-in-phase-1-2-astra-study-for-stargardt-disease-therapy-sb-007-splicebio-