SpliceBio lands $135M for a new kind of eye gene therapy
SpliceBio, a Spanish biotechnology company working on next-generation gene therapies, said Wednesday it raised $135 million in a Series B round to advance its lead program in Stargardt disease into further clinical testing.
The startup is built around a technology that’s meant to overcome a key limitation of current gene therapies. Scores of gene therapy developers use adeno-associated viruses, or AAVs, to deliver genetic cargo into the body. But because of their small size, AAVs can’t carry larger corrective genes, leaving many diseases out of reach or forcing companies to come up with creative solutions.
One example is in Duchenne muscular dystrophy. Because of the large size of the gene coding for the muscle-protecting protein dystrophin Duchenne patients lack, several companies, like Sarepta Therapeutics, have developed gene therapies that make a shortened form. The approach comes with drawbacks, however, as a truncated version of the protein might not work as well as the original.
SpliceBio has a different workaround. The company is using two separate AAVs to send pieces of a large gene into cells. Once inside, specially engineered splicing molecules help reassemble their cargo into full-length proteins.
Stargardt disease, for example, is caused by mutations to the ABCA4 gene, which is too large to stuff into an AAV. SpliceBio says its method can help the body produce a functional version of the protein that gene produces. Its lead program, SB-007, is in a Phase 1/2 trial.
SpliceBio isn’t the only biotech pursuing this type of approach to treat Stargardt, an inherited eye condition that causes progressive vision loss. Another European company, AAVantgarde, has a similar type of program in early-stage testing.
SpliceBio’s technology is based on research conducted at Princeton University, where co-founder and CEO Miquel Vila-Perelló was studying protein design and engineering. Launched in 2014 under the name ProteoDesign, the biotech is also working on additional programs in ophthalmology, neurology and other undisclosed therapeutic areas.
Its Series B round was co-led by EQT Life Sciences and Sanofi Ventures, and involved seven other backers including Roche Venture Fund, New Enterprise Associates and Novartis Venture Fund.
“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 said in a statement. The company raised €50 million in a Series A round in 2022.
Gene and cell therapy startups backed by the roughly two dozen investment firms BioPharma Dive tracks have secured a little over $1.1 billion in venture dollars so far in 2025. Half of those fundings have come in the form of “megarounds” exceeding $100 million apiece, continuing an ongoing trend.
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