In March 2026, news about a gene therapy for epilepsy made headlines around the world. An early trial showed zorevunersen could reduce seizures in children with Dravet syndrome by up to 91%. What’s more, the gene therapy was well-tolerated, with mild-to-moderate side-effects. The research team (led by our President, Professor Helen Cross) are continuing to explore this potentially life-changing treatment in future phase 3 trials.
Zorevunersen is just one example of exciting advances in gene therapy – treatments aiming to counteract or fix faults in the DNA code. Gene therapies are being explored for many conditions and diseases, from haemophilia to cancer to arthritis.
The hope for epilepsy is that these treatments could help address epilepsy that is resistant to existing anti-seizure medications. It might even be possible in some cases to tackle the root cause of the epilepsy and stop seizures for good.
However, as is often the case with big news stories, along with the excitement comes misinformation. The truth is that, while gene therapies are an exciting prospect, a lot more research is needed to bring them into mainstream treatment for some epilepsies.
In this article, we examine some of the myths you might encounter around gene therapy and provide you with the truth – as well as some reasons to be hopeful for the future of epilepsy treatment.
A common myth is that gene therapies change the DNA in every one of your cells, forever.
The reality is that gene therapies work in a much more controlled way. Many gene therapies are based upon adeno-associated viral vectors (AAVs). These harmless viruses can’t replicate by themselves, and do not integrate their genetic material into a person’s DNA code.
They can also be engineered to target only specific cells. This targeted approach is important for future gene therapies for epilepsies, as it means that the treatment is focused on the seizure-causing parts of the brain, rather than the whole body. This hopefully reduces the risk of treatment side-effects.
Dr Yichen Sophie Qiu worked on an even more targeted gene therapy, which switches on only when brain neurones become overactive and switches off when calmed down. Dr Qiu explains more about her Institute-supported PhD research in this article.
While there is a lot of hope for gene therapies, claims of them being ‘cures’ for any disease, least of all epilepsies, are premature.
Because they are so new, not enough time has passed to allow researchers to fully understand how long the effects of these treatments last.
However, it is true that gene therapies aim to do more than just control seizures. Instead, we hope they will address the underlying genetic cause, providing long-lasting seizure reduction. Dr Gareth Morris is developing such a gene therapy for drug-resistant epilepsy with support from the Epilepsy Research Institute, to restore the brain’s natural ability to resist seizures.
Like any treatment, gene therapies do come with side-effects.
The recent results from the early trial of zorevunersen for Dravet syndrome showed the most common side-effect, experienced by 25% of participants, was post-lumbar puncture syndrome (severe headaches that develop after the drug is injected directly into the spinal fluid).
Again, because not enough time has passed, researchers don’t know the potential for long-term side-effects of gene therapies. Ongoing monitoring of people who have received gene therapies will be needed.
One option to mitigate these long-term effects is to develop gene therapies that are active in the brain only for a short, defined period – something that Dr Jenna Carpenter is doing for Dravet syndrome, with support from the Epilepsy Research Institute.
After hearing about successful gene therapies, it’s natural to wonder whether anyone with epilepsy could benefit.
However, the reality is the gene therapies that are being developed at the moment are targeting particular groups of patients. These treatments can be gene-specific, such as the recent zorevunersen trial which targeted a variant of the SCN1A gene. Or they can be anatomy-specific, such as a new treatment called EPY201 which is delivered directly to the seizure-causing area in the brain.
Trials also have strict criteria for entry, such as age or previous treatments received. The benefits and risks of treatment vary across different groups of people.
This means it’s likely that, in the future, there won’t be one ‘silver bullet’ to treat all patients, but a range of options which can be carefully selected for individuals.
The current excitement around gene therapies for epilepsy is justified; they have real potential to tackle the underlying causes of seizures. Advances like these remind us of the power of research to make a tangible difference to people’s lives. However, in all the excitement, we shouldn’t forget the long road that lies ahead to bring these treatments to everyone who could benefit.
The Epilepsy Research Institute will continue to invest in and advocate for gene therapies which can help everyone live free from seizures.
Find out more about the ‘Advanced Therapeutics & Disease Modification’ theme of the Epilepsy Research Institute’s work, and how we’re helping to deliver treatments that could stop epilepsy for good.
