RARE Daily

SpliceBio and Spark Collaborate to Develop a Gene Therapy Targeting an Inherited Retinal Disease

October 17, 2023

Rare Daily Staff

Genetic medicines biotech SpliceBio signed an exclusive collaboration and licensing agreement with Spark Therapeutics to utilize SpliceBio’s proprietary protein splicing platform to develop a gene therapy for an undisclosed inherited retinal disease.

Under the terms of the agreement, SpliceBio and Spark will conduct a research collaboration utilizing SpliceBio’s proprietary Protein Splicing platform, which offers the potential to address 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 commercialize 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.

“This research collaboration and license agreement is an opportunity to develop a novel gene therapy in an area of high unmet medical need,” said Miquel Vila-Perelló, CEO and co-founder of SpliceBio. “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 for patients in need.”

Federico Mingozzi, chief scientific and technology officer of Spark Therapeutics, which is a subsidiary of Roche, said that the company’s gene therapy Luxturna demonstrated the impact gene therapies can have on the lives of patients with inherited retinal disease, but that there are many more people still in need of treatment options.

“With our complementary capabilities, combined deep technical knowledge and SpliceBio’s impressive protein splicing platform we hope to make further progress in the treatment of inherited retinal diseases, bringing new gene therapies into the clinic and to commercial availability,” he said.





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