Gene therapy has capacity issues. The engineered viruses used to deliver these therapies have limited space to carry genetic cargo. SpliceBio, which is developing technology to deliver larger genes, has raised 50 million euros to support its research, including its first goal: gene therapy for a rare inherited eye disease.
This A round The financing announced Wednesday was co-led by UCB Ventures and Ysios Capital.
Gene therapy typically reaches its cellular destination via adeno-associated virus (AAV). While these viral vectors remain the delivery vehicle of choice, their capacity limitations keep some diseases out of reach. Barcelona-based SpliceBio aims to deliver larger genetic cargoes by splitting loads. Biotechnology accomplishes this through a family of proteins called inteins. These proteins are responsible for protein splicing, a biochemical process that involves the cleavage and formation of peptide bonds.
Inteins found in nature are not suitable for human therapy. SpliceBio engineers proteins for protein splicing. According to the company, these engineered introns are capable of splicing proteins, which are then recombined to full-length in vivo. The startup’s technology is based on research from Princeton’s Muir Lab.
SpliceBio’s lead program targets Stargardt disease, a rare eye disease that causes macular degeneration, the macula that is part of the retina. It is caused by a mutation in the ABCA4 gene, which is too large to be carried by a single AAV vector. SpliceBio said it will use the Series A cash to advance its Stargardt disease gene therapy for human testing. While ophthalmic applications are the startup’s primary focus, the company says its technology has been validated in “several other organs beyond the retina.”
The Princeton spin-off was originally named protein design, and its initial focus was on cancer. In 2020, start a business Renamed to Splice Bio After raising seed funding from Ysios and Asabys Partners. (This year appears to have changed the company’s name to a single word.) Asabys also invested in the latest financing announced Wednesday, which SpliceBio calls the largest Series A round for a Spanish biotech. Other new investors in the round include New Enterprise Associates, Gilde Healthcare and Novartis Venture Fund.
“We are proud to have been involved with the company from its inception and have been impressed by the progress made so far,” said Ysios managing partner Joël Jean-Mairet in a prepared statement. “SpliceBio’s platform represents a Unprecedented opportunity to expand the range of diseases that can be addressed through gene therapy. This financing also demonstrates the growing potential of the Barcelona Biotechnology Center.”
Photos by Flickr users Jorge Franganillo through Creative Commons license
