NEW YORK – SpliceBio on Tuesday said it has signed an exclusive collaboration and licensing agreement with Spark Therapeutics, under which the biotech firm will use SpliceBio's proprietary protein splicing platform for gene therapy development.
Spark, a Philadelphia-based subsidiary of Roche, and SpliceBio, a genetic medicines firm, will use the platform to develop, manufacture, and commercialize a gene therapy for an undisclosed inherited retinal disease. Under the terms of the agreement, SpliceBio is eligible to receive upfront, opt-in, and milestone payments up to $216 million, plus royalties on net sales.
Barcelona-based SpliceBio's technology is designed to address diseases that cannot be treated with adeno-associated virus vector-based gene therapies because the targeted gene is too large. The platform is based on an engineered form of inteins, a family of proteins that carry out protein splicing, developed by the firm's cofounders at Princeton University.
"We are proud that Spark Therapeutics recognizes the potential of our pioneering protein splicing platform and the profound impact it could have in the treatment of inherited retinal diseases that are unable to be effectively addressed by other gene therapy approaches," SpliceBio CEO and Cofounder Miquel Vila-Perelló said in a statement.