SpliceBio Announces U.S. FDA IND Clearance of SB-007 to Commence Phase 1/2 Clinical Study in Patients with Stargardt Disease

SB-007 is the only IND-cleared, clinical-stage therapeutic addressing the root cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations
Phase 1/2 ASTRA study set to begin in the first half of 2025, supported by POLARIS, a pioneering company-sponsored natural history study in Stargardt disease
First ever IND clearance for a Protein Splicing gene therapy

BARCELONA, SPAIN, 12 December 2024 – SpliceBio, a genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today 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.

SB-007 is the only IND-cleared, clinical-stage therapeutic addressing the root cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations

Phase 1/2 ASTRA study set to begin in the first half of 2025, supported by POLARIS, a pioneering company-sponsored natural history study in Stargardt disease

First ever IND clearance for a Protein Splicing gene therapy

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