Spain’s SpliceBio will use $135 million from a series B round backed by Sanofi and Roche to advance its lead gene editing therapy for Stargardt disease through clinical studies.
Stargardt disease causes vision loss in children and adults, and SpliceBio’s adeno-associated viral (AAV) gene therapy SB-007 is already undergoing a phase 1/2 trial for the indication. The series B round was co-led by new investors EQT Life Sciences and Sanofi Ventures with support from Roche Venture Fund, New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund and Asabys Partners.
As well as fund the phase 1/2 study of SV-007, which is designed to produce a functional copy of the full-length ABCA4 protein, the biotech said it would also use the series B proceeds to “accelerate” its pipeline of AAV gene therapy programs in ophthalmology, neurology and “other undisclosed indications.”
“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,” SpliceBio CEO Miquel Vila-Perelló, Ph.D., said in a June 11 release.
“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,” Vila-Perelló added. “We are building a company positioned to lead the next wave of genetic medicines.”
Roche’s Spark Therapeutics unit previously penned a $126 million alliance with SpliceBio back in 2023 to use the Barcelona-based biotech’s platform, which aims to sidestep a common challenge in gene therapy where the necessary gene is too large to fit within traditional AAV vectors.
In this morning’s release, Carole Nuechterlein, head of Roche Venture Fund, said the fund had been “impressed by the … momentum behind SB-007 in Stargardt disease, and the platform’s potential to unlock a new class of genetic medicines.”
Splice emerged in 2022 with 50 million euros ($57 million) to tackle delivery barriers in gene therapy, backed by UCB Ventures and the Novartis Venture Fund. The biotech’s work is based on technology developed in the Muir Lab at Princeton University.