Epilepsy gene therapy candidate moves closer to clinical study phase

By Jane Byrne contact

- Last updated on GMT

© GettyImages/Michail_Petrov-96
© GettyImages/Michail_Petrov-96

Related tags: Epilepsy, Gene therapy, Plasmids

Gene therapy developer, CombiGene, and CDMO, Cobra Biologics, have signed off on a deal to secure Good Manufacturing Practice (GMP) production of two essential plasmids needed for the manufacture of CombiGene's gene therapy, CG01.

The gene therapy candidate is designed to treat drug-resistant focal epilepsy.

Jan Nilsson, CEO, CombiGene, said the deal guarantees access to crucial components for the production of CG01, and, in that way, the Swedish company is taking another step closer to clinical studies on its innovative treatment. 

Is there a timeline in place in terms of the first clinical trial of CG01?

“We expect to initiate and finalize the pivotal biodistribution and toxicology studies in 2021 and, thereafter, submit a clinical trial application (CTA),”​ Nilsson told BioPharma-Reporter.

AAV based treatment 

CG01 is a breakthrough therapy with potential to dramatically improve the quality of life for a group of epilepsy patients for whom there is currently no effective treatment, says the Lund, Sweden-based, CombiGene.

The biotech uses an adeno-associated viral vector (AAV) to administer a combination of neuropeptide Y (NPY) and its receptor Y2 directly to the part of the brain where the epileptic seizure begins. Preclinical effect studies have been concluded with positive results, according to the Nordic company. 

Epilepsy is a global problem. The disease, as per data from CombiGene, affects an estimated 0.6 to 0.8% of the world’s population. In 2016, there were 5.7m diagnosed epilepsy patients in the US, France, Germany, Italy, Spain, the UK and Japan. About one-third of these patients do not respond to conventional medical treatment. It is this patient group CombiGene intends to help with CG01.

The gene therapy innovator has put the value of the global market for its drug candidate at US$750m to US$1,5bn per annum.

CombiGene also announced this week the availability of "reliable and meaningful"​ assays for manufacturing control of CG01, established through a collaborative effort with CGT Catapult.

Plasmids production

Increasing production of plasmids to large scale and ensuring they are GMP compliant requires management of the scale-up process, said the partners.

Cobra maintains that its long-established plasmid production platform along with in-house expertise will ensure therapeutic grade quality plasmids for CG01 production.

“With an expert team and strong track record, Cobra can provide a full service offering from plasmid through to viral vector production. Whilst it is not ‘unique’, we are probably the only company that can do this in the same production site,”​ Tony Hitchcock, technical director, Cobra Biologics, told us.

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