SpliceBio partners with Spark Therapeutics to develop gene targeting retinal disease

By Isabel Cameron

- Last updated on GMT

© Getty Images
© Getty Images

Related tags retinal diseases Gene therapy Protein

SpliceBio, a genetic medicines company harnessing protein splicing to develop the next generation of gene therapies, has partnered with Spark Therapeutics to develop a gene therapy for an inherited retinal disease.

Under the terms of the agreement, SpliceBio and Spark will conduct a research collaboration utilizing SpliceBio’s proprietary protein splicing platform.

According to both companies, the platform 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 inherited retinal disease that has not yet been disclosed by either company.

In addition, SpliceBio will be eligible to receive upfront, opt-in and milestone payments up to $216 million and royalties on net sales.

Miquel Vila-Perelló, CEO and co-founder of SpliceBio, said: “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. 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 science and technology officer of Spark Therapeutics, added: “This exclusive partnership builds on Spark’s innovative leadership programs in gene therapies for inherited retinal diseases. Our breakthrough gene therapy LUXTURNA demonstrates the ability to transform the lives of patients with biallelic mutations in the RPE65 gene whose physicians have determined their eligibility for treatment, while providing potential alternative treatment options for patients with other inherited retinal diseases."

"With our complementary capabilities, combined deep technical knowledge and SpliceBio’s revolutionary platform capabilities, we aim to further advance progress in the treatment of inherited retinal diseases, bringing new transformational gene therapies into the clinic and eventually to the global market.”

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