Revolutionary treatment

Revolutionary treatment

for Hirschsprung disease

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Via rectal administration, this treatment aims to promote the regeneration of the enteric nervous system which is otherwise missing from the colon of patients, most of them being young children. The therapy is designed to also globally improve intestinal functions in these children, thereby offering significant hope for their well-being.

A pioneering biotechnology company

Based in Montreal, Neurenati Therapeutics is a pioneering biotechnology company developing an innovative treatment for Hirschsprung disease. Nicolas Pilon, an expert in rare diseases, and Rodolphe Soret, a specialist in neuro-gastroenterology, have combined their expertise to develop an innovative treatment for Hirschsprung disease. With the additional support from the serial entrepreneur Maxime Ranger and various experts in medicine, gastroenterology, and rare disease clinical trials, the company is committed to bringing this treatment into clinical practice.

  • Maxime
    Ranger, PhD, MBA
  • Chief Executive Officer,
  • Chairman of the board
  • Nicolas
    Pilon, PhD
  • Chief Scientific Officer,
  • Cofounder Neurenati
  • Rodolphe
    Soret, PhD
  • Director of the board,
  • Cofounder Neurenati
  • Alexandre P.
    Lebeaut, MD
  • Drug Development Advisor,
  • Director of the board
Marielle Cohard-Radice
  • Marielle
    Cohard-Radice, MD
  • Clinical Development Consultant,
  • Director of the board
  • Benoit
    Doré, PhD
  • Portfolio Manager Axelys,
  • Director of the board
Marie-Eve Bordeleau
  • Marie-Eve
    Bordeleau, PhD
  • Senior Director,
  • Pre-Clinical Development
  • Patrick
    Colin, BPharm, PhD
  • Clinical Development,
  • Consultant
Marie-Eve Bordeleau
  • Dana
    Matzen, PhD
  • Senior Business Advisor to the CEO
  • Francois
    Boutin , CPA
  • Acting Chief Financial Officer,
  • Comptinc

Our technology


Hirschsprung disease represents a potentially fatal congenital malformation characterized by the absence of the enteric nervous system in the colon. This condition leads to contraction of the segment lacking the enteric nervous system, causing obstruction due to fecal matter (megacolon). The enteric nervous system plays a crucial role in other gastrointestinal functions, explaining the increased risk of systemic infections from intestinal microbes in children with Hirschsprung disease.

Currently, the standard treatment involves surgical removal of the affected segment, followed by reconnection of the innervated colon to the anus. Unfortunately, post-surgical complications such as fecal incontinence and enterocolitis often result in hospital readmissions and additional surgeries.


© Mayo Foundation for Medical Education & Research


© Trends in Molecular Medecine


Our innovative approach is based on regenerative medicine, offering a non-surgical alternative. We have demonstrated that the Glial cell-Derived Neurotrophic Factor (GDNF) administered via rectal enema stimulates the formation of a new enteric nervous system in the colon of three Hirschsprung disease mouse models. This newly created enteric nervous system significantly improves gastrointestinal functions, prolonging the life of a significant proportion of these mice. Recent data demonstrate that GDNF, in addition to its beneficial effects on the formation of the enteric nervous system, also exerts a positive influence on the overall intestinal ecosystem.

Indeed, it appears to promote improvement in several essential components, such as strengthening the intestine-specific immune system, maintaining the integrity of the intestinal epithelial barrier, and positively modulating the intestinal microbiota. These findings offer promising insights into the overall and multifunctional impact of GDNF on intestinal health, thus providing new avenues for the treatment of Hirschsprung disease and potentially other intestinal pathologies.