Celebrating Canadian Innovation: A New Chapter for Rare Disease Therapies


Of the 7,000 known rare diseases, less than 10 per cent have an effective drug therapy¹. Of these, about 60 per cent are brought to Canada, where it can take up to an additional six years for approval versus the US or Europe².

And then there’s the cost. Approximately 25% of rare disease therapies approved by Health Canada and recommended by Canada’s Drug Agency (CDA) and/or Institut national d'excellence en santé et services sociaux (INESSS) ever get to patients through the public drug plans³.

Our goal at Eder Therapeutics is to improve the quality and access to care for Canadians with rare diseases. That’s why we’re pleased to announce the completion of a licensing agreement with the National Research Council of Canada (NRC) for a made-in-Canada gene therapy under development for lipoprotein lipase deficiency (LPLD), a rare genetic disorder.

 

This gene therapy is part of the NRC’s Cell and Gene Therapy Challenge program, through which researchers are developing innovative technology solutions for cell and gene therapies that target chronic diseases and rare genetic disorders. The LPLD investigational therapy was developed by the NRC under the leadership of Dr. Danica Stanimirovic, in collaboration with Dr. Michael Hayden and Dr. Colin Ross from the University of British Columbia.

This agreement complements our previously acquired intellectual property rights in gene identification and diagnostics for lipid disorders from LIPIMED and Dr. Daniel Gaudet. Our research and development, including work on the gene replacement therapy, will be conducted in partnership with ECOGENE-21, a renowned Canadian biocluster with a robust clinical research infrastructure and advanced R&D capabilities. ECOGENE-21 specializes in rare diseases or severe common diseases, particularly in the areas of lipid diseases and associated cardiometabolic risks.

Canada has long been a hub for scientific excellence, and this partnership exemplifies our unwavering belief in the power and potential of Canadian innovation. We look forward to working with the NRC and our respective partners to advance therapies that will help improve the lives of people living with rare disease.

REFERENCES

  1. Government of Canada. Building a National Strategy for High-Cost Drugs for Rare Diseases: A Discussion Paper for Engaging Canadians. Available at https://www.canada.ca/en/health-canada/programs/consultation-national-strategy-high-cost-drugs-rare-diseases-online-engagement/discussion-paper.html. Last accessed June 18, 2024.

  2. Canadian Organization for Rare Disorders. About Cord - Key Facts. Available at https://www.raredisorders.ca/about-cord/. Last accessed June 18, 2024.

  3. Canadian Organization for Rare Disorders. Rare Disease in Bill C-64: Similarities, Deviations, and Learnings. Available at https://www.raredisorders.ca/content/uploads/Final-CORD-Pharmacare-Bill-C64-Submission-May-22-2024.pdf. Last accessed June 18, 2024.