Neurenati Therapeutics is a Québec-based biotech focused on rare pediatric diseases, with a lead program targeting Hirschsprung disease (HD). Its lead asset, NEU-001, is a first-in-class, once-in-a-lifetime intrarectal combination therapy using a neurotrophic growth factor intended to regenerate the enteric nervous system and restore gastrointestinal motility in newborns. The company aims to eliminate the need for pull-through surgery, the current invasive and non-curative standard of care. NEU-001 has received U.S. FDA Orphan Drug Designation and Rare Pediatric Disease Designation, which may provide seven years of marketing exclusivity, user-fee exemptions, tax credits, and potential eligibility for a Rare Pediatric Disease Priority Review Voucher upon approval. Neurenati is completing IND-enabling preclinical studies with plans to initiate a first-in-human trial by the end of H1 2026 and is proceeding with cGMP production of the growth factor. The company recently closed a $1.7M extended seed financing to fund development and expand its core team, and appointed Dr. Meriam Kabbaj as Chief Development Officer to lead R&D and regulatory strategy. Neurenati Therapeutics is a Québec-based biotech focused on developing therapies for rare pediatric diseases, with its lead program NEU-001 targeting Hirschsprung disease. NEU-001 is positioned as a growth-factor therapy intended to treat newborns with HSCR and avert the need for surgery and associated complications. The company says HSCR affects roughly 1,300 newborns in North America each year. Recent financing is intended to fund development of an optimal formulation and to accelerate progress toward clinical trials. Neurenati targets filing a clinical trial application in Canada and the United States by the end of 2025. The company is also actively looking for additional technologies in the rare-disease pediatric space. Neurenati Therapeutics is a Québec-based biotech developing NEU-001, a first-in-class growth-factor therapy intended to promote enteric nerve cell formation and treat Hirschsprung disease (HSCR) in newborns. The company aims to offer a non-surgical alternative to current surgical treatment and to reduce lifelong complications such as enterocolitis; HSCR affects roughly 100 Canadian newborns each year. Since 2021 Neurenati holds exclusive worldwide rights to an innovative growth factor technology developed by Nicolas Pilon and Rodolphe Soret at UQAM, and both scientists remain actively involved in the company’s strategy and activities. Neurenati closed a $1.2M seed financing from Genson Capital and has secured a CQDM-SynergiQc grant; Axelys and Quebec’s MEIE provided early support. The seed round remains open and the company expects additional funding during 2024. Neurenati plans to file an IND application for NEU-001 in the second half of 2025 and has added Alexandre Lebeaut as an independent director to its board.

Total raised

$4M

Funding rounds

3

Latest round

Seed

Latest activity

Mar 2025