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NEU-001 (Université du Québec à Montréal)
- Development stage
- Preclinical
- Lead developer
- Neurenati Therapeutics
- Modality
- Small Molecules, Recombinant Proteins and Enzymes
- Administration
- Rectal
01
Overview
**NEU-001** is an investigational fixed-dose combination of recombinant human glial cell line-derived neurotrophic factor and butyrate developed by Neurenati for Hirschsprung disease. Administered intrarectally as a single-cycle treatment, it is designed to activate GDNF-RET signaling, induce enteric neurogenesis in aganglionic colon, regenerate the enteric nervous system, and restore gastrointestinal motility in newborns, potentially avoiding pull-through surgery. The program remains in IND-enabling preclinical development and has received FDA Orphan Drug and Rare Pediatric Disease designations and EMA Orphan Medicinal Product Designation for Hirschsprung disease. ([biospace.com](https://www.biospace.com/press-releases/neurenati-announces-its-first-scientific-and-clinical-advisory-board-meeting-with-kols-to-advance-the-development-of-neu-001-a-promising-cure-for-hirschsprung-disease))
- Other names
- GDNF + butyrateglial cell line-derived neurotrophic factor + butyrate
02
Targets
GDNF (Glial cell line-derived neurotrophic factor)RET (Rearranged during transfection receptor tyrosine kinase)GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)
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