RSQ-SOD1

RSQ-SOD1 (also known as RSQ-330) is a preclinical-stage cyclic peptide macrocycle being developed by ResQ Biotech for the treatment of superoxide dismutase 1 (SOD1)-linked amyotrophic lateral sclerosis (ALS), including both familial and mechanistically related forms. Discovered using the company's proprietary living cell-based discovery platform (CycloFOLDER), RSQ-SOD1 is designed to directly target, rescue, and prevent the misfolding and aggregation of mutant SOD1 proteins. By inhibiting the formation of toxic SOD1 aggregates, the therapeutic candidate aims to mitigate motor neuron degeneration and neurotoxicity. Preclinical studies in patient iPSC-derived motor neurons and C. elegans models have demonstrated its ability to rescue SOD1 misfolding and improve motor function, with in vivo mouse efficacy studies planned to support its advancement toward clinical development.

Other names

SOD1C5-4SOD-1C5-4SOD 1C5-4