Lund-based gene therapy company CombiGene continues to advance its main project CG01 ahead of the phase I studies scheduled to begin next year. The company is now ready for GMP production of drug substance and has also begun a collaboration with UK-based Neurochase in order to optimise the administration of the candidate.
Biotech company CombiGene develops gene therapies for severe diseases that currently lack treatment methods or where existing drugs do not provide satisfactory results. The company’s main project, CG01, which is in the late preclinical stages of development, is being developed as a gene therapy drug for the treatment of drug-resistant focal epilepsy. Focal epilepsy is the most common form of epilepsy in adult epileptics and today’s drugs only treat the symptoms without curing the disease.
Optimised administration for tox studies
The company plans to start a phase I clinical trial next year, and, in preparation for that, the company will conduct toxicology and biodistribution studies. Together with UK-based Neurochase, CombiGene has begun work on optimising the administration of CG01. The two companies have initiated a preclinical study in animals to better understand how to inject the candidate in order to ensure that the gene therapy vectors reach the intended part of the brain.
Neurochase is a specialist in administering drugs to different parts of the brain and, according to CombiGene, has extensive experience in administering precisely the type of gene therapy vectors used in CG01. Results from the study are expected at the end of the summer and will be the basis for moving forward with the preclinical program, where toxicology and biodistribution studies await.
Ready for GMP production
Another milestone for the clinical program is the production of a GMP quality batch of CG01. An important step was recently taken together with partner Cobra Biologics, when production and quality assurance of the plasmids to be used in GMP production was completed.
The plasmids were produced by Cobra Biologics in January and February and have since undergone a number of quality analyses. These have shown that the plasmids are of the correct quality, and the production itself has generated material for more batches of CG01 than originally planned.
»The release of the plasmids produced by Cobra means that we will be able to start GMP production of CG01 later in the year. In doing so, we are taking another important step towards the first human study that we plan to start in 2022,« — commented Jan Nilsson, CEO of CombiGene, in a press release in connection with the announcement.
Project financing secured
Earlier this spring, the company also carried out a share issue that added approximately 75 MSEK before issue costs. The addition means that it now has enough capital to carry out the GMP production and the resource-intensive toxicology and biodistribution studies, providing CombiGene with favourable conditions for submitting a CTA application for CG01 in 2022. Read more about the issue here.
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