Barcelona-based SpliceBio has announced the close of a $135 million Series B financing round, marking a pivotal milestone for the clinical-stage genetic medicines company. The round was co-led by EQT Life Sciences and Sanofi Ventures, with significant participation from Roche Venture Fund and all existing investors, including New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners.
The fresh capital will be used to advance SpliceBio’s lead gene therapy candidate, SB-007, which targets Stargardt disease—a rare inherited retinal disorder that leads to progressive vision loss and currently has no approved treatments.
SB-007 is the first dual adeno-associated viral (AAV) gene therapy to receive FDA clearance for clinical development in Stargardt disease and has also secured regulatory approval from the UK’s Medicines and Healthcare products Regulatory Agency.
The ongoing Phase 1/2 ASTRA trial and the observational POLARIS study are both actively recruiting, aiming to evaluate the therapy’s ability to deliver a functional copy of the full-length ABCA4 protein, regardless of the patient’s specific genetic mutation.
SpliceBio’s innovative Protein Splicing platform underpins its approach, addressing a major limitation of traditional gene therapies—the inability to deliver large genes via AAV vectors. By splitting large genes into smaller transgenes, which are then delivered using dual AAV vectors and reassembled inside the cell, SpliceBio’s technology enables the treatment of genetic disorders previously considered untreatable.
In addition to advancing SB-007, the Series B proceeds will accelerate SpliceBio’s broader pipeline of gene therapies in ophthalmology, neurology, and other undisclosed indications. The company’s platform, developed from pioneering research at Princeton University, has attracted the attention of major pharmaceutical partners.
Roche’s Spark Therapeutics previously entered a $126 million collaboration with SpliceBio to leverage its platform for other gene therapy applications.
Leadership at SpliceBio described the financing as a testament to the strength of its programs and the transformative potential of its proprietary technology. Investors echoed this sentiment, highlighting the company’s strong execution and the promise of its approach to deliver best-in-class therapies for diseases that remain without effective treatments.
As SpliceBio continues to expand its clinical and research footprint, the company is positioning itself at the forefront of the next wave of genetic medicines.
