SpliceBio and Spark Therapeutics Partner To Develop Gene Therapy

India Pharma Outlook Team | Wednesday, 18 October 2023

 India Pharma Outlook Team

SpliceBio, a genetic medicines company that uses protein splicing to develop the next generation of gene therapies, announced the signing of an exclusive collaboration and licensing agreement with Spark Therapeutics to develop a gene therapy for an unspecified inherited retinal disease using SpliceBio's proprietary protein splicing platform. SpliceBio and Spark will collaborate on research using SpliceBio's proprietary protein splicing platform, which has the potential to address diseases that are currently untreatable 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 sell a gene therapy developed as a result of this research cooperation , which will target an unnamed genetic retinal condition. SpliceBio will be eligible for up to $216 million in upfront, opt-in, and milestone payments, as well as royalties on net sales. "This research collaboration and license agreement represents an exciting opportunity to develop a novel gene therapy in a field with significant unmet medical need."

"We are honoured 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 ineffectively addressed by other gene therapy approaches," said SpliceBio CEO and co-founder Miquel Vila-Perelló, Ph.D. "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."

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