FDA clears investigational new drug application for Stargardt disease gene therapy

The FDA cleared an investigational new drug application for SB-007, an adeno-associated viral vector gene therapy designed to address the root genetic cause of Stargardt disease, according to a press release from SpliceBio.
The clinical-stage therapeutic, which may have the potential to treat Stargardt disease across all ABCA4 mutations, will be investigated in the phase 1/2 ASTRA study, which will evaluate the safety and efficacy of a single dose of subretinal SB-007. The study is set to begin in 2025.
In March, the company launched the POLARIS trial, a natural history study of Stargardt

The FDA cleared an investigational new drug application for SB-007, an adeno-associated viral vector gene therapy designed to address the root genetic cause of Stargardt disease, according to a press release from SpliceBio.
The clinical-stage therapeutic, which may have the potential to treat Stargardt disease across all ABCA4 mutations, will be investigated in the phase 1/2 ASTRA study, which will evaluate the safety and efficacy of a single dose of subretinal SB-007. The study is set to begin in 2025.
In March, the company launched the POLARIS trial, a natural history study of Stargardt