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From left to right, Gerard Caelles, CBO; Silvia Frutos, Co-founder and CTO; Miquel Vila-Perello, Co-founder and CEO and Aniz Girach, CMO. Image / SpliceBio
 11.06.2025

SpliceBio raises €118 million in the largest funding round in the biotechnology sector in Spain

SpliceBio breaks records with the largest biotechnology funding round recorded in Spain. The startup, headquartered at the Barcelona Science Park, has closed a Series B round of €118 million led by the Dutch firm EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund. The company will use the funds to advance the clinical development of its lead program SB-007 for Stargardt disease, a hereditary retinal disorder caused by mutations in the ABCA4 gene.

The three new investors EQT Life Sciences, Sanofi Ventures, and Roche Venture Fund will join the company’s shareholder base alongside the previous round investors from the €50 million round closed in 2022, which already included New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners.

Founded in 2020 by researchers Silvia Frutos and Miquel Vila-Perelló, SpliceBio will allocate the funding to progress the clinical development of its gene therapy candidate SB-007 for Stargardt disease. This includes the ongoing phase 1/2 intervention study ASTRA and the observational study POLARIS. SB-007 is the first dual AAV-type gene therapy authorized by the U.S. Food and Drug Administration (FDA) to initiate clinical trials in this pathology, and it also has approval from the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).

Stargardt disease is a hereditary retinal disorder caused by mutations in the ABCA4 gene, leading to progressive vision loss culminating in blindness, for which there are currently no approved treatments. SB-007 aims to directly address the underlying genetic cause by expressing a full-length functional copy of the ABCA4 protein, with the potential to benefit all patients regardless of their specific mutation.

In addition to the development of SB-007, the funds will allow accelerating the expansion of the gene therapy portfolio beyond ophthalmology, including programs in neurology and other yet unrevealed indications.

“This funding marks a fundamental milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our product portfolio in ophthalmology, neurology, and other fields,” says Dr. Miquel Vila-Perelló, CEO of SpliceBio. “The support from such high-quality investors underscores the strength of our programs and our unique protein splicing platform, as well as its potential to develop gene therapies for diseases that remain untreatable today. We are building a company positioned to lead the next wave of genetic medicines,” he concludes.

SpliceBio is opening new possibilities in gene therapy by overcoming one of its main limitations: AAV vectors, commonly used to deliver genes, cannot carry genes that are too large. This has prevented the treatment of many genetic diseases requiring genes longer than what these vectors can transport.

To solve this, the company has developed its own technology based on protein splicing, which uses special proteins called inteins, created from research at Princeton University. Thanks to this technology, the gene is split into two parts that are delivered separately into the body via AAV vectors. Once inside the cell, the inteins help join the two halves and reconstruct the full protein needed to treat the disease. This innovative approach enables the treatment of genetic diseases that could not previously be addressed with conventional gene therapy.

“We are proud to support SpliceBio, a pioneer among the next generation of genetic medicine companies. Its protein splicing platform is designed to offer a novel solution to deliver large genes with AAV, one of the most pressing challenges in the field, and exemplifies our commitment to backing transformative science that can significantly benefit patients’ lives,” explains Daniela Begolo, General Manager of EQT Life Sciences.

Meanwhile, Laia Crespo, partner at Sanofi Ventures, points out that “by enabling the delivery of large and complex genes through its novel protein splicing technology with AAV vectors, SpliceBio has the potential to make a significant impact in gene therapy and provide best-in-class therapies to patients”.

» Link to the news: web de SpliceBio [+]