NEU-001

**NEU-001** is Neurenati's preclinical fixed-combination regenerative therapy for **Hirschsprung disease**. It combines recombinant human glial cell line-derived neurotrophic factor with butyrate and is intended for single-cycle intrarectal administration to newborns and young children. The program is designed to mobilize and differentiate tissue-resident enteric neural progenitor cells, regenerate the absent enteric nervous system in aganglionic colon, and restore gastrointestinal motility, potentially avoiding pull-through surgery. Neurenati reports that NEU-001 improved survival, enteric neurogenesis, and colonic motility in the Holstein mouse model of Hirschsprung disease. ([neurenati.com](https://www.neurenati.com/en/technology/))

Other names

GDNF + butyrateglial cell line-derived neurotrophic factor + butyrate