SB-007 works by delivering the full-length ABCA4 gene, which is intended to reinstate the native ABCA4 protein expression in the retina. Credit: PeopleImages.com – Yuri A/Shutterstock.

The US Food and Drug Administration (FDA) has granted clearance to SpliceBio’s investigational new drug (IND) application to begin the trial of a protein splicing gene therapy, SB-007, designed for Stargardt disease treatment.

It is the first to receive this clearance and is poised to address the genetic root cause of the disease, potentially benefiting all subjects regardless of their ABCA4 mutation.

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The company is set to commence the Phase I/II ASTRA trial in the first half of next year.

This study aims to assess the efficacy and safety of a single subretinal dose of the therapy in subjects with this condition.

It follows the launch of the POLARIS trial in March this year, a natural history study by SpliceBio to monitor the progression of the disease, refine endpoints, and establish streamlined criteria for the upcoming ASTRA study.

A dual adeno-associated virus (AAV) gene therapy, SB-007 works by delivering the full-length ABCA4 gene, which is intended to reinstate the native ABCA4 protein expression in the retina.

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Demonstrating pharmacological activity in animal models and safety in non-human primates, SB-007 has also secured orphan drug designation in both the US and Europe.

SpliceBio CEO and co-founder Miquel Vila-Perelló said: “The FDA IND clearance of SB-007 is a significant achievement for SpliceBio and Stargardt disease patients. 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.

“SB-007 is an adeno-associated viral (AAV) vector gene therapy aimed at restoring expression of the full-length ABCA4 protein, and the only clinical-stage therapy with the potential to help all Stargardt patients.

“We look forward to accelerating the clinical development of SB-007, building on the orphan drug designation granted by the FDA in 2024, and advancing this potentially life-changing therapeutic for patients with Stargardt disease.”

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