Ophthalmologist examining an older Black, male patient's eyes to carry out retinal imaging to look for diabetic retinopathy.
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SpliceBio has signed a collaboration and licensing agreement with Spark Therapeutics, a member of the Roche Group, to use SpliceBio’s proprietary protein splicing platform to develop a gene therapy for 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.

SpliceBio and Spark will use SpliceBio’s the Protein Splicing platform, which aims 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.

“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,” Miquel Vila-Perelló, PhD, CEO and co-founder of SpliceBio.

“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 Vila-Perelló.

Optogenetics, or the field of targeting genetic causes of blindness, has heated up recently, as evidenced by Ray Therapeutics’ recent raising of $100M for retinosa pigmentosa drug development.

“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.

“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 added.

SpliceBio’s lead program targets Stargardt disease, a genetic eye disease that causes vision loss in children and adults. SpliceBio’s platform is based on technology developed in the Muir Lab at Princeton University.

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