FDA clears investigational new drug application for Stargardt disease gene therapy

Key takeaways:

• SB-007 is designed to treat address the root genetic cause of Stargardt disease.
• A phase 1/2 study investigating the safety and efficacy of the therapy is set to begin in 2025.

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 disease “designed to evaluate disease progression, refine endpoints and streamline eligibility criteria” for the ASTRA study, according to the release.

“The FDA IND clearance of SB-007 is a significant achievement for SpliceBio and Stargardt disease patients,” Miquel Vila-Perelló, PhD, CEO and co-founder of SpliceBio, said in the release. “As the first-ever IND for a protein splicing gene therapy, it is a huge step forward to demonstrate the potential of this new therapeutic modality to address diseases caused by mutations in large genes such as ABCA4.”

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