Cobra Biologics, the gene therapy division of Cognate BioServices, a Charles River Laboratories company, and gene therapy company CombiGene AB, have completed the production and quality assurance of the plasmids to be used in the GMP production of CG01, for CombiGene’s gene therapy for the treatment of drug-resistant focal epilepsy.

The now released GMP plasmids were produced by Cobra in January/February this year. The material has since been quality assured through a variety of analyses. These analyses are now complete, and the plasmids have been released at Cobra and are ready to be used as starting material for the GMP batch that will be used in the first clinical study scheduled to begin in 2022. The analyzed plasmids are now part of a stability study, which is required for the company’s upcoming clinical trial application (CTA).

“The release of the plasmids produced by Cobra means that we will be able to start GMP production of CG01 later this year,” said Jan Nilsson, CEO of CombiGene. “In doing so, we are taking another important step towards the first in human study that we plan to start in 2022.”

Mike Austin, corporate vice president, global operations cell and gene therapy CDMO, Charles River, said, “We are pleased to continue the journey with CombiGene and the release of the GMP plasmids represents a crucial step in the production of CG01. We have a well-established plasmid production platform and in-house expertise in quality control that will ensure the delivery of GMP quality plasmid.”

Unlike many gene therapies, which are developed for the treatment of rare diseases, CG01 caters to a large population of patients. Epilepsy is a major global problem. Every year, approximately 47,000 drug-resistant patients with focal epilepsy are estimated to be added in the US, EU4, UK, Japan and China. CombiGene believes that it is realistic that 10-20% of these patients could be treated with the drug candidate CG01. Assuming, for example, that the therapy cost per patient is somewhere between $134,000 and $200,000 (which compared to approved gene therapy drugs is low), it provides sales between $750-$1,500 million annually.