RGX-400

RGX-400 is a preclinical small molecule drug candidate being developed by RemeGenix, in collaboration with Georgetown University, for the treatment of brain injuries, including traumatic brain injury (TBI). The drug is part of a research program focused on the amyloid precursor protein (APP) processing pathway, which is known to be disrupted following head trauma, leading to the accumulation of neurotoxic amyloid-beta peptides. RGX-400 is designed to modulate or inhibit secretases involved in this pathway, specifically Beta-site APP cleaving enzyme 1 (BACE1), to mitigate secondary neurodegeneration and promote recovery after injury. The candidate emerged from the research of Dr. Mark Burns at Georgetown University Medical Center, whose work highlights the role of BACE1 in the acute and chronic pathological changes following brain trauma.