Montreal, March 26, 2025 – McGill University’s DNA to RNA Initiative (D2R) has awarded over $10 million in funding to support three groundbreaking research projects that will advance RNA-based therapies for cancer and rare genetic diseases. The awards were initially announced at the D2R Research Symposium on March 13, 2025.
D2R’s Translational Impact Research (TIR) funding program supports large-scale, translational research projects that will have a measurable impact on the health of Canadians. By addressing gaps in current treatment options, these projects will accelerate the development of RNA-based therapies and make personalized medicine more accessible for patients with rare diseases and cancer. Each project has received more than $3 million in funding, which includes research and training budgets.
The following projects and their principal investigators have been awarded funding:
- RNA Therapy for a Severe and Ultra-Rare Disease – Carl Ernst (McGill University)
Schinzel-Giedion Syndrome (SGS) is a rare genetic disease leading to multi-system deficits and neurological deterioration. Most people with SGS die in the first decade of life and there is no treatment that targets the underlying cause of disease. This project will develop and manufacture an RNA therapy for SGS, with the purpose of reducing seizure burden to alleviate suffering in children living with the disease. This precision medicine approach will pioneer RNA therapy in a child with SGS at the Montreal Children’s Hospital using a new Health Canada approach for severe and ultra rare disease called the Single Patient Clinical Trial.
“For families affected by Schinzel-Giedion Syndrome (SGS), hope is rare—but this research offers just that. The SGS Foundation is proud to support Dr. Carl Ernst’s vital work, bringing us closer to a treatment that could ease suffering and change children’s lives.” – Nuala Summerfield and Dana Bradley, Trustees of the Schinzel-Giedion Syndrome Foundation
- Innovative RNA Therapies Offer New Hope for Patients Affected by POLR3-Related Leukodystrophy – Geneviève Bernard (Research Institute of the McGill University Health Centre)
POLR3-related hypomyelinating leukodystrophy (POLR3-HLD) is a rare genetic neurodegenerative disorder that predominantly affects children and is characterized by reduced white matter (myelin) production in the brain. This project aims to develop RNA-based therapies for POLR3-HLD. Preclinical testing in cell and mouse models will ensure the safety and efficacy of the treatments and will set the stage for clinical testing in humans.
“We are thrilled to advance our research on POLR3-related leukodystrophy with this D2R Translational Impact Research grant. This funding will accelerate the development of novel RNA-based therapies, bringing us closer to life-changing treatments for patients and families affected by this devastating disease. Through innovative science and strong collaborations, we remain committed to translating discoveries into hope.” – Prof. Geneviève Bernard
- The Jerry Pelletier Initiative: Diagnostic and Therapeutic Innovations for Rare Cancers – Mark Lathrop and Morag Park (McGill University)
This project focuses on rare cancer subtypes that are not well-served by existing personalized treatments. This project uses advanced genomics to better understand the biology of these rare cancers. By identifying key markers and potential treatment targets, the research will help identify possible RNA-based therapies and other treatments tailored to each patient. The research will also create valuable resources for the research community to find new ways to treat these difficult cancers.
“We are very pleased that D2R is supporting this program which unites McGill’s forces in genomics and oncology to provide patients with rare cancers new hope for treatments.” – Prof. Mark Lathrop, Scientific Director, D2R
For more information on these projects, please visit D2R’s funded projects page.