Splicebio raises $135m to advance gene therapy for eye disease
FierceBiotech - 11-Jun-2025SB-007 enters phase 1/2 trials to restore vision in Stargardt disease using novel splicing tech
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Genetic medicines company
SpliceBio is a Barcelona‑based clinical‑stage genetic medicines company that’s pioneering a novel “Protein Splicing” platform—rooted in two decades of intein and protein engineering work from Princeton’s Muir Lab—to enable gene therapies for diseases with full‑length genes too large for standard AAV delivery. Their lead program, SB‑007, uses a dual‑AAV approach to deliver the large ABCA4 gene and is currently in a Phase 1/2 trial for Stargardt disease, a genetic retinal disorder with no approved treatments. Backed by top investors including Sanofi, Roche, EQT, and Novartis, SpliceBio is expanding its pipeline into additional ophthalmology and neurology targets, aiming to build a new class of genetic medicines.
Visit website: https://splice.bio/
Details last updated 24-Jun-2025
SB-007 enters phase 1/2 trials to restore vision in Stargardt disease using novel splicing tech