Roche's Spark adds epilepsy asset to pipeline with $328M CombiGene licensing deal

Roche’s Spark Therapeutics unit is offering up to $328.5 million in biobucks for CombiGene’s gene therapy for epilepsy, which has a specific focus on drug-resistant forms of the condition.

CombiGene will receive $8.5 million upfront and $50 million at preclinical and clinical milestones, plus certain R&D expenses and tiered royalties upon commercialization, according to an Oct. 12 statement

Spark will gain exclusive rights to the Swedish biotech’s CG01 project, a gene therapy under development to treat drug-resistant focal epilepsy. An estimated 47,000 patients have this form of treatment-resistant epilepsy across the U.S., EU, U.K., Japan and China, according to CombiGene’s release.

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

RELATED: FDA committee takes on complex gene therapy safety questions with Novartis' Zolgensma providing lessons learned

Spark’s Chief Business Officer Joseph La Barge said the company was impressed by CombiGene’s gene therapy platform. The company taps a network of external researchers to identify potential therapies and move them toward the clinic. The platform was developed with Cobra Biologics and Viralgen.

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

Spark is looking to follow up on the success of Luxturna, the gene therapy approved for a type of retinal dystrophy. The company has a rich pipeline but has not yet claimed a program in epilepsy. Furthest along in Spark’s pipeline are two potential treatments for hemophilia A and B.