SpliceBio, a company focused on genetic medicines, has secured $135m in Series B funding to advance lead program SB-007 in Stargardt disease.
The financing round was co-led by EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund and existing investors such as New Enterprise Associates, UCB Ventures, and others.
The funds will support the clinical development of SpliceBio’s leading gene therapy candidate, SB-007, for Stargardt disease.
This includes the continuation of the Phase 1/2 ASTRA study and the POLARIS observational study. SB-007 is the first dual adeno-associated viral (AAV) gene therapy approved by the FDA for clinical development in Stargardt disease and has also received clearance from the UK MHRA.
Stargardt disease is a genetic retinal disorder caused by mutations in the ABCA4 gene, resulting in progressive vision loss.
Currently, there are no approved treatments. SB-007 aims to address the genetic cause by producing a functional copy of the ABCA4 protein, potentially benefiting all patients regardless of their specific mutation.
The investment will also accelerate SpliceBio’s pipeline of AAV gene therapies in ophthalmology, neurology, and other areas using their Protein Splicing platform.
SpliceBio co-founder and CEO Miquel Vila-Perelló said: “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.
“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.”
SpliceBio is addressing the challenge of delivering large genes using AAV vectors, which are limited to a packaging capacity of 4.7 kilobases. Many genetic disorders remain untreated due to this limitation.
The company’s Protein Splicing platform, developed with technology from Princeton University, uses engineered proteins called inteins to split genes into smaller transgenes. These are delivered using dual AAV vectors, allowing for the assembly of the full-length protein necessary for treatment once inside the cell.
In conjunction with the funding round, Daniela Begolo from EQT Life Sciences, Laia Crespo from Sanofi Ventures, and Carole Nuechterlein from Roche Venture Fund will join SpliceBio’s Board of Directors.
