EQT Life Sciences is pleased to announce that the LSP 7 fund has invested in SpliceBio. The Spanish biotech company is developing novel therapies for genetic diseases - including ophthalmology, neurology, and other therapeutic areas - by leveraging an innovative intein platform. The platform allows large genes to be split into smaller pieces, delivered separately, and then reassembled to create full-length proteins needed to treat diseases.

The USD 135 million Series B financing round will help fund the Phase 1/2 clinical trial of SpliceBio's lead gene therapy candidate, SB-007, which is being developed to treat Stargardt disease. The round was co-led by EQT Life Sciences and Sanofi Ventures, with participation from new investor Roche Venture Fund, as well as all existing investors: New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners.

Stargardt disease, which leads to progressive vision loss and blindness, is the most commonly inherited condition causing degeneration in the macula, affecting 1 in 8,000-10,000 people. Currently, there are no approved treatments available for Stargardt disease. The disease is caused by mutations in the ABCA4 gene, which result in a dysfunctional ABCA4 protein. SpliceBio's SB-007 uses an innovative Protein Splicing technology, based on a family of proprietary engineered proteins called inteins, originally developed at Princeton University. The approach overcomes the challenge of the ABCA4 gene that is too big for traditional gene therapy delivery methods, thereby enabling the production of a healthy ABCA4 protein directly in the retina. 

This approach has the potential to benefit the entire addressable patient pool, regardless of which of the more than 1,200 known mutations responsible for causing Stargardt disease patients carry. Crucially, the difficulty of delivering large genes is also a common barrier in treating many other genetic disorders, highlighting the broad potential and versatility of SpliceBio's technology beyond Stargardt disease.

"This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology, and beyond," said Miquel Vila-Perelló, Chief Executive Officer and Co-Founder of SpliceBio. "The support from such high-quality investors underscores the strength of our programs and our unique Protein Splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today. We are building a company positioned to lead the next wave of genetic medicines."

Daniela Begolo, Managing Director at EQT Life Sciences who will join the SpliceBio Board of Directors, commented: "We are proud to support SpliceBio, a pioneer among the next generation of genetic medicine companies. Its Protein Splicing platform offers a novel solution to deliver large genes, one of the field's most pressing challenges, and exemplifies our commitment to backing science that transforms patients' lives."

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