Â鶹AV
Commercialization Priming
In partnership with Genome Quebec
The Commercialization Priming funding program, in partnership with Génome Québec,Ìýprovides short-term financial support for early-stage efforts that test or validate the commercial potential of well-defined concepts, technologies, or inventions tied to the D2R Initiative’s priority areas.
Applications to this program undergo a comprehensive, multi-step review process. This includes a scientific merit review by external reviewers, followed by reviews from internal and external experts who evaluate both the commercial potential and alignment with D2R. Final approval for co-funding is granted by Génome Québec and D2R’s Research Steering Committee.
| Principal investigator | Name of project |
|---|---|
| Jo Anne Stratton | RNA therapies for the treatment of a rare leukodystrophy |
| Paul Goodyer | An mRNA strategy to restore CTNS expression in Cystinosis |
Funded project summaries
RNA therapies for the treatment of a rare leukodystrophy
ALSP (Adult Leukoencephalopathy with axonal Spheroids and Pigmented glia)Ìýis 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 onÌýthe 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.Ìý
Principal Investigator: Jo Anne Stratton (Â鶹AV)
Co-Investigator(s): Roberta La Piana (Â鶹AV), Thomas Durcan (Â鶹AV)
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) forumlation 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.Ìý
We will answer key questions about the design of therapeutic CTNS mRNAs and the novel LNP formulations that target the kidneys.Ìý
Principal Investigator: Paul Goodyer (Research Institute of the Â鶹AV Health Centre)
Co-Investigator(s): Elena Torban (Research Institute of the Â鶹AV Health Centre)
Project duration: 12-months
Relevant D2R Axes: RNA Therapeutics (Axis 2)
