SpliceBio Doses First Patient in Trial for Stargardt Gene Therapy SB-007
The ASTRA and accompanying POLARIS studies are actively enrolling patients.
This article originally appeared on our sister site, Ophthalmology Times Europe®.
Barcelona-based genetic medicines company SpliceBio has dosed the first patient in the phase 1/2 ASTRA clinical trial (NCT identifier pending), which is evaluating SB-007, a dual adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease.
An inherited disorder caused by mutations in the ABCA4 gene, Stargardt is a progressive retinal dystrophy that currently has no approved treatments available. The disease affects approximately 1 in 8,000 to 10,000 people, making it the most common form of inherited juvenile macular degeneration.
“Stargardt disease is a devastating inherited retinal disorder with no approved treatments available,” principal investigator Paul Yang, MD, PhD, chief of the Paul H. Casey Ophthalmic Genetics Division at Oregon Health & Science University Casey Eye Institute, said in a statement.1 “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.”
SB-007, an investigational protein splicing dual AAV gene therapy, is designed to restore expression of the native full-length ABCA4 protein in the retina. In both Europe and the United States, SB-007 has received orphan drug designation. SpliceBio also continues to enroll patients in the POLARIS study (NCT06435000), a natural history study for Stargardt disease.
“Stargardt disease has remained elusive to gene therapies due to the large size of the ABCA4 gene," Miquel Vila-Perelló, PhD, the chief executive officer and cofounder of SpliceBio, added, explaining why the first dosing in the ASTRA study is such a significant milestone. "SB-007 is the first gene therapy in clinical development designed to restore expression of the full-length ABCA4 protein across all Stargardt disease patients, regardless of their mutations." SpliceBio leadership indicated that the company will share data at scientific conferences as the ASTRA and POLARIS studies progress.
