SpliceBio and Spark to Develop Gene Therapy for Inherited Retinal Disease

SpliceBio announced an exclusive collaboration and licensing agreement with Spark Therapeutics to develop a novel gene therapy for an undisclosed inherited retinal disease, using SpliceBio's proprietary Protein Splicing platform.

In accordance with the terms of the agreement, SpliceBio and Spark will engage in a research collaboration using SpliceBio's proprietary Protein Splicing platform. This platform holds the potential to target diseases that are currently beyond the reach of gene therapies due to the substantial size of the required genes, which cannot be effectively delivered using adeno-associated virus (AAV) vectors.

Spark will hold exclusive worldwide rights for the development, manufacturing, and commercialization of a gene therapy emerging from this research collaboration, focusing on an undisclosed inherited retinal disease. SpliceBio stands to receive upfront payments, opt-in payments, and milestone payments totaling up to $216 million, in addition to royalties based on net sales.

“This research collaboration and license agreement is an exciting opportunity to develop a novel gene therapy in an area of high unmet medical need. 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,” said, Miquel Vila-Perelló, Ph.D., Chief Executive Officer 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.”

“This partnership builds on Spark’s leadership in gene therapies for inherited retinal diseases. Our breakthrough gene therapy LUXTURNA® demonstrated how we can change the lives of patients with biallelic mutations in the RPE65 gene whose physicians have determined their eligibility for treatment. At the same time, there are many more people with other inherited retinal diseases that need treatment options,” said Federico Mingozzi, PhD, Chief Science & Technology Officer of Spark Therapeutics. “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.”