Epilepsy Project CGO1 progressing through optimization activities in preparation for in-human studies

In June, CombiGene and Spark Therapeutics held a bi-annual Joint Research Committee meeting in Stockholm, Sweden, to make a detailed plan for the upcoming six months. Significant progress has been made in the gene therapy field since CG01 was first designed, both in terms of manufacturing and in the formulation of  gene therapies. For the epilepsy project CG01, this means that now is the perfect time to take full advantage of this new and important knowledge and optimize some of the components of the project before moving it towards clinical studies.

Using the combined strength of CombiGene and Spark, the two companies are now engaged in optimization of the therapeutic AAV to achieve improved coverage of the brain regions affected by epilepsy.

“The Joint Research Committee meeting in Stockholm was highly productive, and again proved that we have an ideal partner in Spark”, says Karin Agerman, Chief Scientific Officer at CombiGene. “I am really happy about the decision to optimize some of the components of the project. By doing this, we will be in a much more advantageous position once the project enters in-human studies,” Karin concludes.

About CG01 
CG01 is a unique gene therapy candidate aimed at a large patient population to solve an unmet need in epilepsy treatment. Epilepsy is a major global medical problem with approximately 47,000 drug-resistant patients with focal epilepsy estimated to be added each year in the US, EU4, UK, Japan, and China. CG01 is in a late preclinical stage, and the production platform, jointly developed by CombiGene and its partners Charles River (previously Cobra Biologics Ltd.) and Viralgen, satisfies all quality requirements and is scalable to meet all future needs, from limited volumes for preclinical and clinical trials to full commercial production.

About Spark Therapeutics 
Spark Therapeutics is a fully integrated, commercial company committed to discovering, developing, and delivering gene therapies. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by disease. For more information, visit www.sparktx.com, and follow us on LinkedIn.