Biotechnology company developing precision medicines for severe neurological and neuromuscular diseases
Biotechnology company developing precision medicines for severe neurological and neuromuscular diseases. It uses human genetics and a functional genomics discovery platform based on human cell lines to identify disease targets and develop therapies that address underlying disease biology. Its lead candidate, NIDO-361, was a small-molecule androgen receptor modulator studied for spinal and bulbar muscular atrophy (SBMA); after its Phase 2 trial did not meet expectations, the company announced it,