PHILADELPHIA, January 27, 2023 — Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Neurochase, a company specializing in the development of scalable therapeutic strategies and enabling technology for direct delivery to the central nervous system (CNS), today announced a strategic collaboration to develop Neurochase’s proprietary delivery technology for use with selected gene therapies for rare diseases in CNS. Through this collaboration, Neurochase will bring their deep expertise in direct drug delivery technology to Spark’s leading AAV platform.
“There continues to be high unmet medical need in treating CNS disorders, and at Spark we see the significant potential of gene therapy to provide new treatment options for patients,” said Federico Mingozzi, Chief Science & Technology Officer, Spark Therapeutics. “Neurological conditions are known to be challenging to target and treat, and we look forward to collaborating with Neurochase toward our goal in improving delivery of AAV-gene therapies to the brain. Leveraging our combined expertise further supports Spark’s ongoing research and development of gene therapies for diseases affecting the CNS, like Huntington’s Disease.”
Neurochase is developing a proprietary drug delivery system and this collaboration enables Spark to develop selected gene therapies for CNS disorders using the Neurochase technology, which aims to improve targeted delivery of AAV gene therapy to neural structures using the method of Convection Enhanced Delivery (CED).
“Applying CED-based delivery systems for AAV gene therapies is an emerging strategy for the treatment of neurological disorders,” said Sharon Kane, CEO, Neurochase. “Advanced delivery systems for AAV gene therapies are promising techniques to allow targeted delivery past the blood-brain barrier with the ability to achieve therapeutic drug concentrations. This collaboration with Spark is an important milestone in our company’s work to improve outcomes for patients and extend tolerability of therapies for central nervous system disorders, including novel gene therapies.”
About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.
Neurochase, founded by Professor Steven Gill, aims to bring transformative therapies to patients with neurological diseases using state of the art technology. Neurochase specializes in direct drug delivery to the CNS and provides globally scalable treatment strategies and solutions for the pharmaceutical and biotech industry. Neurochase Ltd. is a Registered Company Number 12428919; www.neurochase.com.