Novel first-in-class voltage-gated calcium channel positive allosteric modulators that stabilize the open state of the channel and have therapeutic potential to treat a variety of neuromuscular conditions - Sophion

Novel first-in-class voltage-gated calcium channel positive allosteric modulators that stabilize the open state of the channel and have therapeutic potential to treat a variety of neuromuscular conditions


Author(s): Stephen D. Meriney, Peter Wipf, Kristine Ojala, Stephanie Aldrich, Man Wu, Tyler Tarr, Scott Ginebaugh, Mary Liang, Rozita Laghaei, Mary Cheng, Ivet Bahar, Jon Johnson


We have recently developed a series of novel, first-in-class, voltage-gated calcium channel (VGCC) positive allosteric gating modifiers that are selective for the types of VGCCs that regulate transmitter release at neuromuscular synapses (CaV2). These small molecules do not cross the blood-brain barrier well and have been shown to have therapeutic potential to treat a variety of neuromuscular diseases characterized by weakness of presynaptic origin (Lambert-Eaton myasthenic syndrome (LEMS) and Spinal Muscular Atrophy (SMA).