Rare Diseases, Neuropathies, and Cancers

Rare Diseases, Neuropathies, and Cancers

Major Funding from Médicament Québec to Unlock the Potential of Therapeutic RNA

The Montreal Clinical Research Institute (IRCM) and its partners at McGill University and the University of Sherbrooke are pleased to announce that they have secured significant funding of $1,296,688 to propel the rollout of an integrated service offering for the development of RNA-based therapies for rare and complex diseases, neurological disorders, and cancers.

Granted by Médicament Québec, this funding is allocated to the DePTAQ Network (Réseau de Développement et de Production de Thérapies ARN du Québec), a cutting-edge, transformative initiative in the biomanufacturing of RNA therapies that brings together the leading expertise in RNA and the synergy of three academic platforms from major biomedical research institutions in Québec:

  • The IRCM with its therapeutic RNA platform, led by Dr. Martin Sauvageau, Director of the RNA and Noncoding Mechanisms of Disease Research Unit at the IRCM.
  • The University of Sherbrooke with its RNomics platform, led by Dr. Benoit Chabot, a professor and leading expert in alternative RNA splicing and its roles in cancer, apoptosis, and aging.
  • And McGill University with its mRNA Therapeutics Platform, led by Dr. Thomas Duchaine, Professor and Chair of the Department of Biochemistry and founding Director of the McGill Centre for RNA Sciences.

Supported also by the Quebec Consortium for Drug Discovery (CQDM) and the Ministry of Economy, Innovation, and Energy (MEIE), the DepTAQ network aims to transform Quebec’s RNA scientific excellence into a sustainable operational infrastructure capable of supporting the maturation of innovations, from discovery through to preclinical validation of therapeutic RNAs. The inclusion to the project of industrial partners such as Galenvs Sciences, NemRod, NumBio Therapeutics, Element Biosciences, Oxford Nanopore Technologies, and New England Biolabs, brings key expertise and will enable procedures to be tested under realistic and transferable conditions, while strengthening local supply chains—a top strategic objective.

Why is this important?
RNA splicing is a key mechanism in the production of proteins that ensures the proper functioning of body cells, each according to its specific role. However, several studies show that alterations in the splicing of certain RNAs lead to the production of defective or toxic proteins that can disrupt the regulation of the nervous system and cause neurological diseases (neuropathies). Moreover, mutations affecting splicing can cause rare diseases such as leukodystrophies, due to the absence or defectiveness of the proteins produced. It has also been shown that abnormal splicing is a major characteristic of cancers, promoting tumour growth and the spread of metastases.

RNA-based therapies hold great potential for effective and promising innovation regarding these diseases, against which modern medicine often has few answers. In this context, driving inter-institutional and synergistic initiatives such as DePTAQ is crucial to supporting the research ecosystem toward the development of concrete and innovative therapeutic options.

At the IRCM, we firmly believe in the potential of the RNA revolution, and we are working on multiple fronts to develop innovative and effective therapies. We are glad about this funding, which will serve as a catalyst for this initiative. More than just a scientific project, it represents a strategic investment that strengthens Quebec’s position as a competitive hub of innovation in the field of RNA. – Dr. Jean-François Côté, President and Chief Scientific Director of the IRCM.

Accelerating the Development of Therapies
With this major funding, DePTAQ’s partner platforms will be able to develop unique processes and conduct high-value-added proof-of-concept scientific studies, particularly by leveraging lessons learned from pilot projects focused on conditions such as leukodystrophies and neuropathies caused by RNA splicing defects. 

A complementary component will focus on modulating splicing to enhance the replication of oncolytic viruses as part of an anti-cancer approach. Using cellular models, research teams will identify splicing abnormalities caused by genetic mutations and explore potential solutions. This work will serve both as a proof of concept and as a springboard for the development of personalized therapies.

Furthermore, under the leadership of Dr. Panagiotis Prinos, Director of DePTAQ, this structural funding will enable the establishment of a quality management system grounded in robust shared infrastructure and the implementation of standardized operating procedures with sustainable operational foundations, in order to meet the specific needs of partners in the biomedical research ecosystem (clinical, academic, and industrial sectors).

This project will thus serve as a solid testing ground and a springboard for future personalized RNA-based therapies developed in Quebec.

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