ADB-104 is our development program for the treatment of Focal Refractory Epilepsy. Our novel CA8* gene therapy, activating Kv7, is designed to locally target specific brain cells after precise mapping of the epileptic locus (‘hot spot’) and treatment using stereotaxic injection (a precise minimally invasive procedure performed by neurosurgeons) into the seizure locus and decrease neuronal excitability in this location without ablation or surgical excision.

Focal Refractory Epilepsy (FRE)

FRE arises from a specific site in the brain (the “hot spot”)^1,2.  This specific type of epilepsy is comorbid with anxiety, depression, and social stigma³.  FRE is very difficult to treat with medications and is most-often drug-resistant. Current treatment options involve anti-epileptic medications, laser ablation or brain tissue excision² to reduce the seizure burden and preserve long-term brain function.

• About 10 million worldwide with focal refractory epilepsy⁵
• About 2 million US, UK, EU patients with FRE^5​

Limited ​treatment options

• Anti-epileptic medications often fail and have serous side effects⁶
• Surgery (brain resection or laser ablation) often complicated by AEs^7,8

1. Nascimento et al (2023): https://doi.org/10.1002/epd2.20045
2. Kwan et al (2010): https://doi.org/10.1111/j.1528-1167.2009.02397. (remove x)
3. Scott et al (2017): https://doi.org/10.1111/epi.13769
4. https://www.who.int/news-room/fact-sheets/detail/epilepsy
5. Sources for prevalence estimates: CDC; Epilepsy Society; (remover V)
6. Perucca et al (2012): https://doi.org/10.1016/S1474-4422(12)70153-9
7. Lamberink et al (2020): https://doi.org/10.1016/S1474-4422(20)30220-9
8. Chen et al (2023): https://doi.org/10.1111/epi.17560