CG01

**CG01** is a gene therapy drug candidate developed by CombiGene AB for the treatment of drug-resistant focal epilepsy, a condition affecting millions worldwide where up to 30% of patients do not respond to standard anti-epileptic drugs. It utilizes an adeno-associated virus (AAV) vector to deliver genes encoding human **neuropeptide Y (NPY)** and its **Y2 receptor** into brain cells, upregulating these proteins to inhibit epileptic seizures, as demonstrated in preclinical studies. The therapy aims for a potential one-time curative treatment rather than lifelong symptomatic relief, with a scalable GMP production process established via partners like Cobra Biologics and Viralgen; it received EU Horizon 2020 funding and was exclusively licensed to Spark Therapeutics (Roche group) in 2021, though the partnership ended due to Spark's pipeline reprioritization, leaving CombiGene seeking new partners while confident in its preclinical data.[1][2][3][4][5]