平特五不中
Commercialization Priming
In partnership with Genome Quebec
The Commercialization Priming funding program, in partnership with G茅nome Qu茅bec,听provides 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鈥檚 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 G茅nome Qu茅bec and D2R鈥檚 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)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 (平特五不中)
Co-Investigator(s): Roberta La Piana (平特五不中), Thomas Durcan (平特五不中)
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.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 平特五不中 Health Centre)
Co-Investigator(s): Elena Torban (Research Institute of the 平特五不中 Health Centre)
Project duration: 12-months
Relevant D2R Axes: RNA Therapeutics (Axis 2)
