NBI-921352, a first-in-class, NaV1.6 selective, sodium channel inhibitor that prevents seizures in Scn8a gain-of-function mice, and wild-type mice and rats.

doi:10.7554/eLife.72468

Article

NBI-921352, a first-in-class, NaV1.6 selective, sodium channel inhibitor that prevents seizures in Scn8a gain-of-function mice, and wild-type mice and rats.

eLife - 2 Mar 2022

Johnson J P, Focken Thilo, Khakh Kuldip, Tari Parisa Karimi, Dube Celine, Goodchild Samuel J, Andrez Jean-Christophe, Bankar Girish, Bogucki David, Burford Kristen, Chang Elaine, Chowdhury Sultan, Dean Richard, de Boer Gina, Decker Shannon, Dehnhardt Christoph, Feng Mandy, Gong Wei, Grimwood Michael, Hasan Abid, Hussainkhel Angela, Jia Qi, Lee Stephanie, Li Jenny, Lin Sophia, Lindgren Andrea, Lofstrand Verner, Mezeyova Janette, Namdari Rostam, Nelkenbrecher Karen, Shuart Noah Gregory, Sojo Luis, Sun Shaoyi, Taron Matthew, Waldbrook Matthew, Weeratunge Diana, Wesolowski Steven, Williams Aaron, Wilson Michael, Xie Zhiwei, Yoo Rhena, Young Clint, Zenova Alla, Zhang Wei, Cutts Alison J, Sherrington Robin P, Pimstone Simon N, Winquist Raymond, Cohen Charles J, Empfield James R

Abstract excerpt

NBI-921352 (formerly XEN901) is a novel sodium channel inhibitor designed to specifically target NaV1.6 channels. Such a molecule provides a precision-medicine approach to target SCN8A-related epilepsy syndromes (SCN8A-RES), where gain-of-function (GoF) mutations lead to excess NaV1.6 sodium current, or other indications where NaV1.6 mediated hyper-excitability contributes to disease (Gardella and Møller, 2019;...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

NBI-921352, a first-in-class, NaV1.6 selective, sodium channel inhibitor that prevents seizures in Scn8a gain-of-function mice, and wild-type mice and rats.

Abstract excerpt

Topics

Related research