SpliceBio SL, a clinical-stage genetic medicines company in Spain, has received $135 million in Series B financing to advance a new therapy for Stargardt disease, an inherited condition that can lead to the progressive loss of vision and blindness. The financing was co-led by EQT Life Sciences and Sanofi Ventures, with participation from the Roche Venture Fund and existing investors. It will help finance an ongoing Phase 1/2 trial of the company’s lead gene therapy candidate SB-007.
There are currently no approved therapies for Stargardt disease but multiple research projects are underway, one of which is a small molecule vitamin A substitute designed to slow the progression of the disease. SpliceBio said its programme is intended to advance the science by addressing the genetic cause of the disease.
Stargardt arises from mutations in the ABCA4 gene which provides instructions for making protein in the retina, a light-sensitive tissue that lines the back of the eye. Mutations in the gene cause vision disorder which worsens over time. SB-007 is a functional copy of the ABCA4 gene that is delivered to the eye in an adeno-associated virus vector (AAV). Owing to the size of the gene relative to the chosen vector, the company has used proprietary technology to split the gene into two transgenes. The transgenes are then delivered using dual vectors.
Once inside the cell, the DNA of each transgene is transcribed into messenger RNA and translated into a full-length protein reportedly with the potential to treat all patents irrespective of their specific genetic mutation. In a statement issued on 11 June, Daniela Begolo, managing director of EQT Life Sciences, said the new technology “offers a novel solution to deliver large genes, one of the field’s most pressing challenges.”
The funding will support the interventional Phase 1/2 trial as well as a corresponding observational study. The candidate therapy has received orphan designations from both the US Food and Drug Administration and the European Medicines Agency. SpliceBio’s pipeline includes gene therapy programmes across several therapeutic areas including ophthalmology and neurology. The platform technology was developed in the Muir laboratory at Princeton University, US.
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