Roche’s Spark adds epilepsy asset to pipeline with $ 328 million CombiGene license agreement


Roche’s Spark Therapeutics unit is providing up to $ 328.5 million in biobucks for CombiGene’s gene therapy for epilepsy, which specifically focuses on drug-resistant forms of the disease.

CombiGene will receive $ 8.5 million upfront and $ 50 million at preclinical and clinical stages, as well as some R&D expenses and tiered royalties upon commercialization, according to an Oct. 12 report. declaration.

Spark will obtain the exclusive rights to the CG01 project from Swedish biotech, a gene therapy in development to treat drug-resistant focal epilepsy. An estimated 47,000 patients have this treatment-resistant form of epilepsy in the US, EU, UK, Japan and China, according to the CombiGene statement.

The companies have been in touch as CG01 moves through the final stages of preclinical development, CombiGene CEO Jan Nilsson said in the statement.

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Spark Commercial Director Joseph La Barge said the company was impressed with CombiGene’s gene therapy platform. The company uses a network of external researchers to identify potential therapies and move them to the clinic. The platform was developed with Cobra Biologics and Viralgen.

CombiGene is also working on the CGT2 project, which aims to develop gene therapy for partial lipodystrophy, a rare metabolic disease.

Spark seeks to build on the success of Luxturna, the approved gene therapy for a type of retinal dystrophy. The company has a rich pipeline but has yet to claim an epilepsy program. The most advanced in Spark’s pipeline are two potential treatments for hemophilia A and B.


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