SpliceBio has secured $135 million in Series B funding to further the clinical development of its gene therapy SB-007 for Stargardt disease and to expand its therapeutic pipeline.

Target Information

SpliceBio, a cutting-edge biotechnology company, has successfully closed a Series B funding round of $135 million. This funding will primarily support the clinical development of its lead program, SB-007, which targets Stargardt disease, a hereditary retinal disorder caused by mutations in the ABCA4 gene. The company employs a novel Protein Splicing technology designed to deliver the ABCA4 gene using a dual adeno-associated virus (AAV) vector, overcoming significant limitations imposed by the conventional AAV delivery methods that can accommodate only smaller gene sizes.

Currently, there are no approved treatments for Stargardt disease, which leads to progressive vision loss and blindness. SB-007 aims to restore the production of the ABCA4 protein within the retina, potentially benefiting all Stargardt patients regardless of their specific ABCA4 mutation. With the successful financing, SpliceBio is poised to not only advance SB-007 but also expand its pipeline in gene therapies across ophthalmology, neurology, and other therapeutic areas.

Industry Overview

The biotechnology industry globally, and specifically within the United States, is undergoing rapid transformation, particularly in the area of gene therapy. As demand for innovative healthcare solutions increases, more funding is being