Announcement – Rare Disease Day

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CQDM and Neurenati Therapeutics Fund the Development of an Innovative Treatment against Hirschsprung Disease

Montreal, February 29, 2024 – On this International Rare Disease Day, CQDM is proud to announce the funding of a collaborative research project aimed to develop a revolutionary therapeutical approach for Hirschsprung disease. This project with a total value of over $1.7 million is made possible by a grant from the ministère de l’Économie, de l’Innovation et de l’Énergie of the Government of Québec (MEIE) awarded by CQDM of $766 039 and a contribution from Neurenati Therapeutics.

Hirschsprung disease is a rare, life-threatening congenital malformation in which the enteric nervous system is absent or deficient in the colon, driving its obstruction by faeces. The only available treatment is based on surgical removal of the diseased segment, but post-surgery problems are frequent and often require hospital readmission and a new surgery procedure. Several surgery patients will later be affected by enteric colitis and other intestinal transit complications as they age.

The Québec company Neurenati Therapeutics, co-directed by Maxime Ranger and Rodolphe Soret, and a research team lead by Professor Nicolas Pilon, director of The Center of Excellence in Research on Orphan Diseases – Fondation Courtois (CERMO-FC), and Professor Lekha Sleno at UQAM, identified a growth factor called GDNF. The latter can induce new enteric nervous system formation in the colon, offering hope of an effective therapeutic solution for patients, while avoiding surgery.

This collaborative project will validate the basis of a new treatment for Hirschsprung disease with a non-surgical approach based on regenerative medicine. By the end of this project, Neurenati Therapeutics aims to have elucidated the mechanism of action of GDNF-based combination therapies. Moreover, the team will have identified predictive biomarkers in response to treatment, which are essential data for future clinical trials and will place Québec as a leader in treatments for rare diseases.

Our government firmly believes that innovation and collaborative research can enhance Quebecers’ quality of life. We support researchers and companies operating in the life sciences sector. They are working tirelessly to develop promising therapeutic approaches”, said Pierre Fitzgibbon, Minister of Economy, Innovation and Energy, Minister responsible for Regional Economic Development and Minister Responsible for the Metropolis and the Montreal Region.

CQDM is proud to contribute to the development of an innovative approach to meet the long-ignored needs of patients suffering from rare diseases, like the Hirschsprung disease. This project brings a lot of hope to the patients, in addition to enabling the young emerging Québec company Neurenati Therapeutics to benefit from collaborative research and development to validate the foundations of its main interest therapy. A fine example of collaboration that consolidates Québec’s expertise in innovative treatments for rare diseases.” said Fanny Guimont-Desrochers, Director, Scientific Affairs of CQDM.

About Neurenati Therapeutics

Neurenati Therapeutics, co-funded by the researchers Rodolphe Soret and Nicolas Pilon, is a start-up company dedicated to the development of a non-surgical treatment against Hirschsprung disease, a rare condition impacting enteric nervous system in colon of newborns. This treatment, based on regenerative medicine approach, find its origin in works of Professor Pilon, conducted at his laboratory at UQAM associated with The Center of Excellence in Research on Orphan Diseases – Fondation Courtois (CERMO-FC). Proof of concept were established in murine models and patient tissues demonstrating extremely promising results. This treatment implicates repeated rectal administration of a formulation containing a growth factor to stimulate and support the development of the enteric nervous system, which is otherwise absent from the colon of affected children.

About CQDM

Facilitator of biopharma innovation

CQDM is a not-for-profit biopharmaceutical research consortium whose mission is to support and facilitate multi-stakeholder collaborative research and development aimed at accelerating the translation of innovative technologies into solutions that address unmet medical needs, while generating significant benefits for the Quebec and Canadian economy. For more information, please visit the website: www.cqdm.org

Media Contact:

Carl Baillargeon

Senior Director – communications and marketing

CQDM

514 926-9196

cbaillargeon@cqdm.org

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