SpliceBio, a genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, announced that the U.S. Food & Drug Administration (FDA) has cleared its investigational new drug (IND) application for lead program SB-007. 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. SpliceBio is a member of CATALONIA.HEALTH.
Miquel Vila-Perelló, 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.”
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.
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