Development of manufacturing process for novel gene therapy

CombiGene and Cell and Gene Therapy Catapult (CGT Catapult) have signed an agreement to develop a manufacturing process for a unique gene therapy to treat epilepsy

CGT Catapult and CombiGene announced they will collaborate on a project to develop manufacturing processes for CombiGene's drug candidate CG01, a novel gene therapy for the treatment of epilepsy.

The collaboration aims to develop a complete and final manufacturing process, which will allow CombiGene to progress to commercial GMP production and thereafter clinical trials.

CombiGene is pioneering a new therapy with the potential of dramatically improving the quality of life for a group of epilepsy patients for whom there currently is no effective treatment available.

CombiGene’s unique platform uses gene therapy vectors to deliver a combination of neuropeptide y (NPY) and NPY receptors into brain cells, which has shown to inhibit epileptic seizures, in a series of preclinical studies. 

“I am very proud and very pleased to be working with CGT Catapult,” said Jan Nilsson, CEO of CombiGene.

“CGT Catapult is highly renowned for its cutting-edge competence and state-of-the-art infrastructure for advanced therapeutic medicinal products. We evaluated several potential collaborators and we could not have chosen a better partner.”

In addition to CGT Catapult’s capabilities in a range of critical areas such as manufacturing development and regulatory support, the organisation offers the possibility for its partners to commit to the individual steps in a development process one by one, which is very important for CombiGene.

“CombiGene is a small gene therapy company, from a financial and funding point of view, the step-by-step approach is very attractive for us,” said Arne Ferstad, Chairman of CombiGene.

In 2017, CombiGene finalised two important studies with highly promising results. Initial data from the preclinical proof-of-concept-study, showed that CombiGene’s candidate drug, CG01, reduces the frequency of epileptic seizures in animals. Final data from the study will be presented during the first quarter of 2018.

Initial data from the second human expression study, showed that the therapeutic genes encoded by CG01 are expressed in epileptic human tissue, thereby confirming that this method of administering genes encoded by the candidate drug is successful in human tissue.

“We are delighted to be working with CombiGene to accelerate the commercialisation of an important gene therapy to treat an unmet medical need. It is testament to our international reputation and the capabilities that we offer that we continue to be the development partner of choice for innovative cell and gene companies.” said Keith Thompson, CEO of CGT Catapult.

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