The Translational Impact Research (TIR) funding program supports large-scale translational research projects in RNA-based therapeutics, which address D2R鈥檚 strategic priorities, and have a measurable translational impact on the health of Canadians.

In the second funding cycle launched in 2025, 11 Letters of Intent were received of which four were invited to submit full applications. Four full applications were received and awarded. View a summary of the review and selection process.

Funded project听summaries

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From Trial to Gene Therapy: Restoring Differentiation in IDH-Mutant Glioma

IDH-mutant gliomas exhibit incomplete and transient responses to IDH inhibition due to failure of malignant cells to fully exit stem-like states. This project integrates a window-of-opportunity clinical trial with spatial multi-omics, patient-derived explants, and preclinical models to define mechanisms of resistance to IDH inhibitors in vivo. Building on these insights, we will test two RNA-based strategies - LNP-mediated CDKN2A restoration and antisense repression of SOX4 - to reinforce differentiation and overcome resistance. Together, this work establishes a patient-to-therapy pipeline for RNA-based precision treatment of brain cancer.

Principal investigator:听Charles Couturier听(91丝瓜视频)

Co-investigators:听Kevin Petrecca (91丝瓜视频), Jerome Fortin (91丝瓜视频), Carl Ernst (91丝瓜视频)

Partner Organizations:听Servier Canada and Montreal Neurological Insitute-Hospital

Award duration: 3 years

Relevant D2R axes: RNA Therapeutics (Axis 2) Clinical Research, Acceleration, and Implementation (Axis 4),听Data Science, Bioinformatics, and Computing in Personalized Medicine (Axis 5)

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mRNA therapy for CLN3 Batten disease

Batten disease is a devastating neurodegenerative disorder in children with no effective treatment. One type of Batten's disease is caused by complete loss of a gene called CLN3. If the gene can be delivered to the brain of affected children before they become symptomatic, the disease might be stopped before it begins which is usually around the ages of 8-10. In this project, we will assess the stability and biodistribution of synthetic CLN3 RNA encapsulated in a lipid nanoparticle delivered to the spinal canal of mammals. This work could provide a new therapy for people with CLN3-Batten disease.

Principal investigator:听Carl Ernst (91丝瓜视频)听

Award duration: 3 years

Relevant D2R axes: Population Studies and Genomic Medicine (Axis 1), RNA Therapeutics (Axis 2), Clinical Research, Acceleration, and Implementation (Axis 4)

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From Bench to Bedside: RNA and AAV Gene Therapies for Diffuse Midline Gliomas

Diffuse midline gliomas are deadly brain tumors that mainly affect children and young adults. We showed that these tumors are driven by an abnormal protein that keeps tumor cells stuck in an immature, stem-like state, preventing normal brain development. Removing this abnormal protein stops tumor growth in experimental models. We will build on these discoveries by developing new gene-targeting therapies to switch off this driver and by using targeted viral approaches to kill tumor cells and stimulate the immune system. Working with national clinical networks, we aim to rapidly move these treatments toward clinical trials for affected children in Canada.

Principal investigator:听Nada Jabado (91丝瓜视频 - Research Institute of the 91丝瓜视频 Health Centre)听

Co-investigators:听Loic Binan (91丝瓜视频), Claudia Kleinman (91丝瓜视频), Basile Tessier Cloutier (91丝瓜视频), Jason Karamchandani (91丝瓜视频), Darcy Wagner (91丝瓜视频)

Partner Institutions: Trogenix Ltd, United Kingfom

Award duration: 3 years

Relevant D2R axes: Population Studies and Genomic Medicine (Axis 1), RNA Therapeutics (Axis 2), Bioprocessing, Biomanufacturing, and Nanotechnology听(Axis 3), Clinical Research, Acceleration, and Implementation (Axis 4),听Data Science, Bioinformatics, and Computing in Personalized Medicine (Axis 5), Ethical, Socioeconomic, and Cultural Dimensions in Genomic Research (Axis 6)听

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LNP/mRNA Therapy for Sickle Cell Disease

Sickle cell disease (SCD) is a severe inherited disease that impairs oxygen-carrying red blood cells (RBCs) and predominantly affects people of African descent, with the existing therapies being either ineffective or inaccessible. RBCs are uniquely suitable for mRNA-based therapy, and our team has validated a novel method for mRNA delivery to the RBC-precursors. Here we therefore develop and evaluate novel mRNA-based therapies in preclinical models of SCD, including mice, human stem cells, and patient samples. With the clinical team, industry partners, and patient organizations, our goal is to develop an effective and widely accessible therapy for SCD.

Principal investigator:听Anastasia Nijnik (91丝瓜视频)听

Co-investigators:听Paul Goodyer (91丝瓜视频)

Partner Organizations:听Nanofacile Inc.,听Sickle Cell Disease Association of Canada (SCDAC), and Association d'Anemie Falciforme du Quebec (AAFQ)

Award duration: 3 years

Relevant D2R axes: RNA Therapeutics (Axis 2), Bioprocessing, Biomanufacturing, and Nanotechnology (Axis 3), Clinical Research, Acceleration, and Implementation (Axis 4)

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