SpliceBio raises US$ 135M to advance eye disease gene therapy

SPAIN – SpliceBio, a biotech company from Spain, has raised $135 million in a Series B funding round to support its mission of treating inherited eye diseases, starting with Stargardt disease, a rare condition that leads to vision loss in both children and adults.

Founded in 2020, SpliceBio is not your typical gene therapy company. It’s one of the few using a powerful approach called Protein Splicing, which helps deliver large genes that current delivery systems like AAV vectors can’t handle on their own.

Traditional AAV gene therapies have a size limit, which leaves many serious genetic diseases untreated because their corresponding genes are just too big.

SpliceBio’s solution is to split the gene into parts, deliver them separately into the body, and then use engineered proteins called inteins to stitch them together inside the cell—like molecular sewing machines.

The company’s leading therapy, called SB-007, is the first dual-vector AAV gene therapy approved by both the FDA and the UK’s MHRA for clinical trials in Stargardt disease.

It aims to replace the faulty ABCA4 gene, the root cause of the disease, with a working version. This could offer hope to thousands who currently have no treatment options.

The new funding will support two key studies: the ASTRA Phase 1/2 clinical trial, which evaluates the safety and early effectiveness of SB-007, and the POLARIS observational study, which gathers crucial data on patients living with Stargardt disease.

The money will also help SpliceBio expand its gene therapy programs into other areas like neurology and undisclosed diseases.

Investors in this round include notable names such as EQT Life Sciences, Sanofi Ventures, and Roche Venture Fund, alongside existing supporters including Novartis Venture Fund, UCB Ventures, and NEA.

These groups are betting on SpliceBio’s potential to rewrite the rules of gene therapy by unlocking diseases that were previously considered untreatable.

The science behind SpliceBio originated over 20 years ago in the Muir Lab at Princeton University, where researchers investigated inteins and their potential applications in protein engineering.

That deep research foundation is now powering a company with global ambitions to transform the way gene therapies are developed and delivered.

Miquel Vila-Perelló, SpliceBio’s CEO and co-founder, called the funding “a pivotal milestone,” and thanked investors for backing their platform, which he believes can “lead the next wave of genetic medicines.”

With competition heating up—other biotech firms, such as AAVantgarde, are working on similar therapies—SpliceBio’s substantial clinical progress and unique platform position it at the forefront of addressing one of gene therapy’s most significant limitations.