Synthetic engineered potassium channel (EKC) for gene therapy

Available For: Licensing

Summary

Epilepsy affects around 60 million people globally and there remains an urgent need for novel treatment methods. Researchers at UCL Institute of Neurology have developed a novel synthetic engineered potassium channel (EKC) as a gene therapy tool to treat epilepsy and other neurological conditions. The inventors have preclinical results demonstrating their engineered potassium channel reduces seizures in a rodent model of epilepsy.

The Technology and its Advantages

It is estimated that, of the 60 million people who suffer from epilepsy globally, approximately 30% do not respond to pharmacotherapy. Furthermore, surgical intervention by removal of seizure-generating tissue is not suitable for 90% of cases and is limited by damage to normal brain function. Gene therapy offers a promising avenue to address these challenges to treating epilepsy.

Potassium channels reduce the propensity of neurons to fire and to release neurotransmitters. This makes them attractive as a mechanism for treating epilepsy and other neurological conditions that involve excessive firing of neurons, such as chronic pain. Evidence suggests that introducing potassium channels to the brain using viral vectors can be effective in dampening neuronal excitability and treating experimental epilepsy in rodents.

Researchers at the UCL Institute of Neurology have designed a novel expression vector comprising an engineered gene that encodes an edited potassium channel. The modifications to the gene result in enhanced translation and activity of the potassium channel, as well as improved detection of gene expression in a cell. Furthermore, there is improved safety by using a tissue-specific promoter that allows targeted expression of the potassium channel at excitatory neurons only, thereby limiting off-target effects.

This EKC approach uses non-integrating lentiviral particles to deliver the optimised gene construct with preclinical results demonstrating this tool reduces seizures in a rodent model of epilepsy.

Market Opportunity

The global epilepsy market was estimated at $4.2 billion in 2012 and is predicted to reach $5.35 billion by 2022 (GlobalData). The market is currently saturated with over 20 marketed antiepileptic drugs (AEDs), many of which are available in generic form. They all have undesirable side effects and have limited efficacy when treating refractory epilepsy. There is therefore an unmet need for therapies with novel mechanisms of action that address the shortfall of current AEDs.

Intellectual Property Status

UK priority patent application filed.

Further Information

Please contact Derek Reay, Senior Business Manager |T:+44 (0)20 7679 9000 |E: d.reay@uclb.com

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