SpliceBio Initiates $216 Million Collaboration with Roche’s Gene Therapy Unit to Pave the Way for Innovative Inherited Retinal Disease Treatment
SpliceBio, headquartered in Barcelona, is embarking on a transformative partnership with Roche’s gene therapy subsidiary, Spark Therapeutics, aimed at revolutionizing the treatment of inherited retinal diseases. This pioneering collaboration has the potential to yield $216 million in support for Splice, encompassing an undisclosed upfront payment, opt-in opportunities, significant milestones, and future royalties.
The crux of this partnership hinges on SpliceBio’s proprietary Protein Splicing platform, designed to address a recurring obstacle in gene therapy: the challenge of accommodating large genes within traditional adeno-associated virus vectors. By circumventing this constraint, the Spanish biotech aims to unlock new therapeutic possibilities, extending beyond the scope of current gene therapy solutions.
Spark Therapeutics, an established leader in the gene therapy arena, and now a subsidiary of Roche since its acquisition in 2019 for approximately $4.8 billion, is the ideal partner for SpliceBio. Notably, Spark achieved a significant milestone in December 2017 when it secured approval for Luxturna, the first gene therapy for a hereditary vision loss disorder that could lead to blindness. Despite its integration into the Roche family, Spark continues to operate under its original name, underscoring its commitment to advancing innovative gene therapies.
Earlier this year, Spark Therapeutics sealed a collaborative agreement with Neurochase, emphasizing advancements in delivery mechanisms for gene therapies targeting rare central nervous system disorders.
SpliceBio entered the biotech arena in February 2022, backed by 50 million euros (equivalent to $57 million), with a mission to confront the challenges associated with gene therapy delivery. Supported by prominent investors such as UCB Ventures and the Novartis Venture Fund, SpliceBio’s flagship program focuses on addressing Stargardt disease, an inherited genetic eye disorder responsible for vision impairment in both children and adults.
This collaborative venture between SpliceBio and Spark Therapeutics signifies a promising step toward groundbreaking gene therapies for inherited retinal diseases, offering hope to countless patients and highlighting the potential of cutting-edge technology in the field of genetic medicine.
