SpliceBio has made up a US $216m alliance with Spark Therapeutics

SpliceBio has licenced its intein technlogy to Roche's gene therapy subsidiary Spark Therapeutics to develop a gene therapy targeting an undisclosed inherited retinal disease.

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Under a globally exclusive licence option agreement with Roche AG, SpliceBio secures an upfront payment, opt-in and milestone payments up to $216m plus royalties in exchange to its recombinant split intein-based protein splicing platform. SpliceBios platform offers the potential to address hundreds of rare inherited diseases such as retinal photoreceptor-specific diseases that currently cannot be treated with gene therapies because the necessary gene is too large to be delivered by adeno-associated virus (AAV) vectors.

Spark will have exclusive worldwide rights to develop, manufacture, and commercialise a gene therapy arising from this research collaboration targeting an undisclosed inherited retinal disease. SpliceBio will be eligible to receive upfront, opt-in and milestone payments up to $216 million and royalties on net sales. 

“In addition to the Spark collaboration, we continue to develop our lead program in Stargardt disease and further build our capabilities and pipeline of wholly-owned gene therapy programs to develop life-changing therapies.” said, Miquel Vila-Perelló, Ph.D., Chief Executive Officer and co-founder of SpliceBio.

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