AMT-260

AMT-260 is an investigational, one-time administered in vivo gene therapy designed for the treatment of refractory mesial temporal lobe epilepsy (MTLE). It consists of an adeno-associated virus serotype 9 (AAV9) vector that locally delivers two engineered microRNAs targeting the GRIK2 gene, which encodes the GluK2 subunit of kainate-type glutamate receptors. Aberrant expression of GluK2 in the hippocampus is implicated in seizure activity among patients with drug-resistant MTLE. By silencing GRIK2 via miRNA-mediated knockdown, AMT-260 aims to reduce or eliminate seizures. Preclinical studies demonstrated dose-dependent reductions in seizure frequency and significant knockdown (>90%) of GluK2 expression with a favorable safety profile. The therapy is currently being evaluated in a Phase I/IIa clinical trial (GenTLE) for adults with unilateral refractory MTLE[1][3][4][5][6].