SpliceBio Raises $135 Million Series B to Advance Gene Therapy for Stargardt Disease Using Novel Protein Splicing Platform
核心洞察
SpliceBio (搜索) secured $135 million in Series B funding co-led by EQT Life Sciences (搜索) and Sanofi Ventures (搜索) to advance its lead gene therapy candidate SB-007 for Stargardt disease (搜索).
The company's innovative Protein Splicing platform overcomes the challenge of delivering large genes by splitting them into smaller pieces that reassemble into full-length proteins.
SB-007 targets Stargardt disease (搜索), the most common inherited macular degeneration (搜索) affecting 1 in 8,000-10,000 people with no approved treatments currently available.
Spanish biotech company SpliceBio (搜索) has completed a $135 million Series B financing round to advance its lead gene therapy candidate SB-007 for Stargardt disease (搜索), a genetic eye disorder that causes progressive vision loss and blindness. The round was co-led by EQT Life Sciences (搜索) and Sanofi Ventures (搜索), with participation from new investor Roche Venture Fund and all existing investors including New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners.
Addressing an Unmet Medical Need
Stargardt disease (搜索) represents the most commonly inherited condition causing degeneration in the macula, affecting 1 in 8,000-10,000 people worldwide. Currently, no approved treatments are available for this progressive condition that leads to vision loss and blindness in both children and adults. The disease stems from mutations in the ABCA4 (搜索) gene, which result in a dysfunctional ABCA4 protein essential for retinal function.
Innovative Protein Splicing Technology
SpliceBio (搜索)'s approach centers on its proprietary Protein Splicing platform, which utilizes engineered proteins called inteins originally developed at Princeton University (搜索). This technology addresses a fundamental challenge in gene therapy: the ABCA4 (搜索) gene is too large for traditional gene therapy delivery methods.
The platform works by splitting large genes into smaller pieces that can be delivered separately and then reassembled to create full-length proteins needed to treat diseases. For SB-007, this enables the production of a healthy ABCA4 (搜索) protein directly in the retina, potentially benefiting patients regardless of which of the more than 1,200 known mutations they carry.
Clinical Development and Broader Pipeline
The Series B proceeds will primarily fund the Phase 1/2 clinical trial of SB-007, marking a critical milestone in the company's development trajectory. Beyond Stargardt disease (搜索), SpliceBio (搜索) is expanding its pipeline across multiple therapeutic areas including ophthalmology, neurology, and other genetic disorders (搜索) where large gene delivery presents similar challenges.
"This financing marks a pivotal milestone for SpliceBio (搜索) as we advance the clinical development of SB-007 for Stargardt disease (搜索) and continue to expand our pipeline across ophthalmology, neurology, and beyond," said Miquel Vila-Perrelló, 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."
Platform's Broader Potential
The difficulty of delivering large genes represents a common barrier in treating many genetic disorders (搜索), highlighting the broad potential and versatility of SpliceBio (搜索)'s technology beyond Stargardt disease (搜索). The company's platform is based on technology developed in the Muir Lab at Princeton University (搜索) following more than 20 years of pioneering intein, Protein Splicing, and protein engineering research.
Daniela Begolo, Managing Director at EQT Life Sciences (搜索) who will join the SpliceBio (搜索) 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."
The financing positions SpliceBio (搜索) to advance what the company describes as the next wave of genetic medicines, potentially addressing diseases that have remained untreatable due to the technical limitations of current gene therapy approaches.