SpliceBio has announced that the U.S. Food & Drug Administration (FDA) has cleared its investigational new drug (IND) application for lead program SB-007.
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SB-007 is the only clinical-stage therapeutic addressing the root genetic cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations.
Miquel Vila-Perelló, Ph.D., chief executive officer, and co-founder of SpliceBio, 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.”
“Stargardt disease has been a challenge for the development of gene therapies due to the large size of the ABCA4 gene, and currently has no approved therapies available,” said Professor Paul Yang, M.D., Ph.D., chief of the Paul H. Casey ophthalmic genetics division at Casey Eye Institute at Oregon Health & Science University. “This new therapy utilises a unique approach to replace the full-size, normal ABCA4 protein at high efficiency, which addresses the root cause of Stargardt disease across any pathogenic variant in the ABCA4 gene. This IND clearance represents a major milestone in the field and I am thrilled to be part of the clinical studies exploring this promising approach that could transform the lives of Stargardt disease patients.”
SpliceBio plans to initiate enrolment in the Phase 1/2 ASTRA study in the first half of 2025. ASTRA will evaluate the safety and efficacy of a single dose of SB-007 administered subretinally in patients with Stargardt disease. In March 2024, SpliceBio launched the POLARIS trial, a pioneering company-sponsored natural history study of Stargardt disease designed to evaluate disease progression, refine endpoints, and streamline eligibility criteria for accelerated enrolment into the Phase 1/2 ASTRA study. This study will enable Stargardt disease patients to benefit from more precise diagnoses, more rigorous disease monitoring, and potentially faster access to innovative therapies.