Gene and cell therapies

ETX-101 acts by restoring the inhibitory function of GABAergic interneurons by upregulating the expression of voltage-gated type I sodium channels, which are implicated in the pathology of Dravet syndrome.² It is currently being evaluated in a Phase 1/2 trial.²

References:

1.Shaimardanova AA, Chulpanova DS, Mullagulova AI, et al. Gene and cell therapy for epilepsy: A mini-review. Front Mol Neurosci 2022; 15: 868531.

2.Perucca E, White HS, Bialer M. New GABA-targeting therapies for the treatment of seizures and epilepsy II: treatments in clinical development. CNS Drugs 2023; 37 (9): 781‒795.

3.Quilón PG, Volpedo G, Cappato S, et al. Antisense oligonucleotides as a precision therapy for developmental and epileptic encephalopathies. CNS Neurosci Ther 2024; 30 (11): e70050.