Spark Therapeutics Partners with SpliceBio on a Gene Therapy for Eye Disease

Published on: 

Spark Therapeutics and SpliceBio have formed a collaboration to develop a gene therapy that can treat an inherited retinal disease.

On Oct. 17, 2023, SpliceBio, a genetic medicines company headquartered in Spain, announced that it has signed an exclusive collaboration and licensing agreement with Spark Therapeutics under which SpliceBio will use its proprietary protein splicing platform to develop a gene therapy for treating an inherited retinal disease. Details about the disease target were not disclosed.

Under the agreement, both companies will conduct a research collaboration utilizing SpliceBio’s technology platform. The protein splicing platform offers the potential to address diseases that currently cannot be treated with gene therapies. In those cases, the gene required for the treatment is too large to be delivered by adeno-associated virus (AAV) vectors. With this agreement, Spark will have exclusive worldwide rights to develop, manufacture, and commercialize a gene therapy resulting from this research collaboration; the gene therapy will target an undisclosed inherited retinal disease, according to a company press release. The agreement makes SpliceBio eligible to receive upfront, opt-in, and milestone payments of up to $216 million as well as royalties 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 [p]rotein [s]plicing 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, CEO and co-founder of SpliceBio, in the press release. “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 [voretigene neparvovec-rzyl] 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, chief science and technology officer of Spark Therapeutics, in the release. “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.”

Source: SpliceBio