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Splicebio raises $135m to advance gene therapy for eye disease

SB-007 enters phase 1/2 trials to restore vision in Stargardt disease using novel splicing tech

11-Jun-2025

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Barcelona-based biotech SpliceBio has raised $135 million in a Series B funding round to advance its lead gene therapy, SB-007, which targets Stargardt disease—a genetic eye disorder that causes progressive vision loss in both children and adults. The funding round was co-led by EQT Life Sciences and Sanofi Ventures, with additional backing from major investors including Roche Venture Fund, Novartis Venture Fund, and others. This marks a significant step toward clinical development, with SB-007 already in a phase 1/2 clinical trial.

SB-007 is designed to deliver a full-length version of the ABCA4 protein, mutations of which are responsible for Stargardt disease. The therapy uses adeno-associated viral (AAV) vectors for gene delivery—a common platform in gene therapy. However, SpliceBio’s unique "Protein Splicing" platform addresses one of the biggest challenges in the field: delivering genes that are too large for traditional AAV packaging. The company aims to extend this platform beyond ophthalmology into neurology and other disease areas.

CEO Miquel Vila-Perelló, Ph.D., described the funding as a pivotal moment for SpliceBio, emphasizing that the investment validates the promise of the platform to tackle diseases that have so far remained untreatable. The company, which spun out of research at Princeton University’s Muir Lab, is positioning itself as a leader in the next generation of genetic medicine.

SpliceBio previously entered into a $126 million collaboration with Roche’s Spark Therapeutics in 2023 to apply its platform to other gene therapy challenges. Commenting on the latest funding round, Carole Nuechterlein, head of Roche Venture Fund, praised the progress of SB-007 and the broader potential of SpliceBio’s approach. With this fresh capital, the biotech plans not only to push SB-007 through trials but also to accelerate a wider pipeline of therapies for multiple genetic conditions.

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Roche

Multinational healthcare company that operates under Pharmaceuticals and Diagnostics divisions all over the world

Sanofi

Company dedicated to research and development, manufacturing and marketing of pharmaceutical drugs.

SpliceBio

Genetic medicines company

Topics mentioned on this page:
Investments, Gene Therapy
Splicebio raises $135m to advance gene therapy for eye disease