Nido Bio: treating neurodegeneration with small molecules

Nido emerged from stealth on Monday with $109 million in venture funding and a clinical small molecule corrector of the mutation that causes spinal and bulbar muscular atrophy. The biotech was founded by 5AM Ventures’ 4:59 Initiative and houses two small molecule programs and a stem cell-based platform for target discovery.

Nido Biosciences Inc.’s lead indication, spinal and bulbar muscular atrophy (SBMA), or Kennedy disease, is a genetic disorder caused by a repeat expansion in the gene that encodes the androgen receptor (AR). The mutated gene gets turned into an abnormal receptor protein carrying a polyglutanine expansion at its N terminus, which is prone to aggregate and leads to both a toxic gain of function and reduced sensitivity to androgens. ...