A novel gene therapy for epilepsy

ended

The aim of this project is to clinically validate CG01 as a novel gene therapy for the treatment of drug-resistant epilepsy patients. Epilepsy affects approximately 50 million people worldwide. 6 million Europeans are afflicted by the condition which annually costs around EUR 15 billion in France, Germany and the UK alone. Current standard of care entails anti-epileptic drug (AED) therapy. However, up to 30% of epileptics (20 million people worldwide) are unresponsive. Research to date has shown that adeno-associated virus (AAV) vector-mediated upregulation of the human neuropeptide Y (NPY) and its receptor Y2 inhibits epileptic seizures. This has inspired CG to develop CG01, a type of advanced therapy medicinal product based on the introduction of genes that encode human NPY and Y2 via the AAV vector into the brain cells of epileptic patients. AAV has already been shown as clinically safe and validated gene delivery system. The current project aims to translate promising findings in animal research into valid human data by bringing CG01 through a Phase I/II trial in drug resistant epilepsy patients. This will be the basis for further clinical development and commercialisation of the product. CombiGene’s strategic operations are currently aligned to establish CG01 as a novel epilepsy treatment. This innovation has the potential to revolutionize treatment for epileptics by delivering a treatment that may cure rather than just alleviate symptoms. Developing CG01 is integral to the company’s growth and this project is therefore essential in fulfilling the company’s long term vision.