Funded project summaries

Conversion of a gene therapy into a RNA therapy using a novel nanoparticle system

This project aims to develop a new RNA therapy for the rare neurodegenerative disorder ARSACS, using advanced C-TERP nanoparticles to deliver treatment directly to neural cells. Unlike traditional gene therapies that can cause immune reactions and dosage issues, this RNA-based approach offers better control, repeat dosing, and improved delivery to the nervous system. Building on prior success with gene therapy, researchers will compare RNA and DNA delivery methods to determine the most effective strategy for restoring key proteins and protecting nerve cells. Initial studies will use long-term neural cell cultures, paving the way for future testing in animal models and other related conditions. This technology has broad potential for treating rare genetic disorders, and the collaboration with QurCan positions the team for further funding and industry partnerships, with interest already emerging from major pharmaceutical companies.

Principal Investigator: Gentil Benoit (蘑菇短视频)
Co-Investigator(s): Heather Durham (Montreal Neurological Institute)
Collaborator(s): John Reid (QurCan), Mohammad Amini (QurCan)
Project duration: 12-months
Relevant D2R Axes: RNA Therapeutics (Axis 2)

Poly-NS saRNA dengue vaccine: A novel platform for T cell-driven protection against severe dengue

This proposal outlines a collaborative effort with the start-up Vaccine for Communities (V4C) to advance dengue vaccine development by leveraging cutting-edge self-amplifying RNA (saRNA) technology. Building on V4C鈥檚 intellectual property for the current mRNA vaccine, our laboratory will develop a novel saRNA vaccine and conduct rigorous preclinical testing in validated mouse models of severe dengue disease. The project aims to establish proof of concept for the saRNA platform, demonstrating immunogenicity, protection, and safety through comparative studies with V4C鈥檚 mRNA vaccine and the licensed QDENGA (TAK-003) vaccine. Additionally, this research will lay the groundwork for future intellectual property through improvements to the alphavirus-replicon system, enhancing antigen expression, immunogenicity, and tissue-specific delivery.

Principal Investigator:听Silvia Vidal听(蘑菇短视频)
Co-Investigator(s):听Leo Liu听(蘑菇短视频)
Collaborator(s): Anavaj Sakuntabhai (Institut Pasteur, Tokyo, Japan, and V4C),听Etienne Simon-Lori猫re (Institut Pasteur, Paris, France, and V4C)
Project duration: 12-months
Relevant D2R Axes: RNA Therapeutics (Axis 2)

RNA therapies for the treatment of a rare leukodystrophy

ALSP (Adult Leukoencephalopathy with axonal Spheroids and Pigmented glia)听is a genetic disorder caused by mutations in a gene important for immune cell function, especially important for immune cells in the brain, called microglia. When this gene is not working properly patients have motor and cognitive dysfunction leading to death within 6-7 years of diagnosis. It is clear that microglial activation in ALSP induces increased inflammation leading to brain tissue damage, but the therapeutic options for patients remain scarce and are primarily focused on听the management of symptoms. With this proposal we aim to understand how state-of-the art RNA therapies can be optimized to reverse cytotoxicity of human microglia in ALSP patients and provide a novel and disease-modifying treatment strategy.听

Principal Investigator: Jo Anne Stratton (蘑菇短视频)
Co-Investigator(s): Roberta La Piana (蘑菇短视频), Thomas Durcan (蘑菇短视频)
Collaborator(s): Martin Sauvageau (IRCM)
Project duration: 12-months
Relevant D2R Axes: RNA Therapeutics (Axis 2)

An mRNA strategy to restore CTNS expression in Cystinosis

Cystinosis is a rare autosomal recessive disease caused by mutations of the CTNS gene. Without this gene, infants develop failure to thrive, progressive kidney failure and gradual deterioration of other organs.

Our project will show that a stabilized CTNS mRNA in a new lipid nanoparticle (LNP) formulation can prevent deterioration of the kidneys, when delivered early in life to our Ctns-mutant mice and can reverse kidney damage if given later in life.听

We will answer key questions about the design of therapeutic CTNS mRNAs and the novel LNP formulations that target the kidneys.听

Principal Investigator: Paul Goodyer (Research Institute of the 蘑菇短视频 Health Centre)
Co-Investigator(s): Elena Torban (Research Institute of the 蘑菇短视频 Health Centre)
Project duration: 12-months
Relevant D2R Axes: RNA Therapeutics (Axis 2)