Report
Teaser, summary, work performed and final results
Periodic Reporting for period 1 - CG01 (A novel gene therapy for epilepsy)
Teaser
CombiGene’s CG01 project aims at developing a breakthrough gene therapy for patients with epilepsy for which there currently is no effective treatment available. It is estimated that 5.7 million people in the five largest countries in the EU and in the US, suffer from...
Summary
CombiGene’s CG01 project aims at developing a breakthrough gene therapy for patients with epilepsy for which there currently is no effective treatment available. It is estimated that 5.7 million people in the five largest countries in the EU and in the US, suffer from epilepsy. About one-third of them do not receive sufficient treatment by the available pharmaceuticals and a fraction of these are eligible for surgery. Approximately 1.8 million Europeans with the drug-resistant form of epilepsy are currently suffering from uncontrolled seizures contributing to a significant socioeconomic burden. Therefore, there is still a large unmet need for new anti-epileptic and anti-epileptogenic therapies.
In the first clinical study CombiGene will include patients eligible for surgery and positive results in these patients will open up a possibility to treat a larger group of patients. Successful completion of the CG01 project will mean that people suffering from epilepsy will have access to a therapy that will allow them to live a much more normal life, without the negative effects of epilepsy and without the side effects of the currently available medications. For society, successful completion of the CG01 project will mean an improvement in the quality of life for a large group of people hence, allowing them to fully participate in the working as well as in social life.
The overall objective of the project is to develop an effective, first-in-class gene therapy for epilepsy and lay the ground for future gene therapies for other illnesses.
CombiGene is currently preparing the biodistribution and toxicology studies that are prerequisites for the first clinical study.
Work performed
To date, the project is proceeding overall as planned with a few deviations. The deviations were all solved during the first reporting period. The work in the first half of the project is primarily analytical technology transfer and validation of the research production process in preparation for the production of product required for the toxicology studies. The project is managed by Karin Agerman who coordinates all the work performed by the team and by subcontractors. The quality of the clinical activities (including preparation of the clinical trial) is secured by an on-going scientific and clinical management. A scientific advisory board, assembled in November 2018, evaluated the pre-clinical program, identifying gaps in the current data in preparation for the first clinical study. Additionally, a clinical advisory board met in April 2019, in London, where the clinical study design was reviewed. CombiGene received valuable feedback which will guide us in the planning and execution of the clinical trial. The critical risks affecting the project have been identified, assessed and analysed in order to mitigate the impact and ensure the overall efficiency and continuity of the project activities. In the first reporting period, CombiGene did not encounter any problems related to project management. CombiGene invested in additional human resources, hiring new consultants and strengthening the board with one new team member. Throughout the year CombiGene’s team has been actively disseminating and communicating the results and progress of the work to various groups of stakeholders. Members of the CombiGene team delivered numerous presentations and speeches during side events and scientific conferences.
Final results
The activities of the project fully correspond to the expected impact. CombiGene is developing an innovative solution with a curative intent for epilepsy patients, including drug-resistant patients who have no alternative options to date. The drug candidate, CG01 is positioned to move beyond the current state-of-the-art in epilepsy treatment and establish itself as the first safe and efficacious treatment for patients with epilepsy who are unresponsive to AED treatment. CG01 has the potential to fill the existing treatment gap in the epilepsy market and change the current treatment landscape offering potentially life-changing treatment strategies. CG01’s success will further strengthen the company and facilitate its growth while attracting new investors.
Website & more info
More info: https://combigene.com/en/.