Operating Grant : Bringing Rare Disease Gene Therapies to Clinical Trial Readiness – Full Application

Well-being, Health and Biomedical Discovery


Academic Unit: inquire with unit

Memorial Deadline: Thursday 8th, February 2024

External Deadline: Tuesday 13th, February 2024


Rare diseases (RDs) constitute an under-recognized global health priority. In fact, notwithstanding their name, rare diseases are far from rare. The population prevalence of rare diseases is reported at 3.5-6.0% and they affect as many as 446 million people globally1.

Of the 7,000 known rare diseases, only 5% have a treatment, with far fewer having a cure, and >80% are single gene disorders, many of which are likely amenable to gene therapy approaches. The last five years in the gene therapy field have witnessed the approval of several gene therapies for inherited diseases in Europe, the United States, and Canada. Many gene therapies for rare diseases have the potential to constitute one-time treatments with lifelong curative potential. However, a gap remains in Canada between translating therapies from efficacy demonstration in disease models to human clinical trial readiness.


This funding opportunity will support research to bridge the current gap between model organisms/systems and clinical trials for RD drugs in Canada, ultimately increasing the capacity to bring gene therapies to clinic. Key elements of this research will include:

  • Training and Capacity-Building: This funding opportunity will enhance training support by building capacity among knowledge users, patient(s)/caregiver(s)/family, and researchers from a diversity of population groups and across career stages to move gene therapies to clinical trials. This funding opportunity will foster interdisciplinary, inclusive, equitable and culturally safe training and mentoring environments.
  • Knowledge Mobilization: Research teams will be required to develop a knowledge mobilization plan that outlines the strategies that will be used for uptake of the implementation research undertaken by clinicians, patient groups, and policy makers, as they move toward clinical trial readiness.

Role and Contributions of Applicant Partners: CIHR recognizes that a broad range of partners may be relevant to this opportunity, and it is expected that applicant(s) describe the role of all applicant partners and how/if they will contribute to research and research related activities. Any consideration of risk and/or conflict of interest should also be explained, as appropriate.

  • National Research Council – Human Health Therapeutics (NRC-HHT)

This funding call represents a unique opportunity to maximize the value of various federal investments in preclinical research and biomanufacturing capacity in Canada.

The NRC-HHT is a key resource that is expected to provide in-kind support at the Full Application stage and advise on research methods and approaches, in particular as they relate to compatibility with NRC’s technology solutions for gene therapy and biomanufacturing platform. The NRC-HHT is available to provide their expertise to up to two (2) projects over four (4) years that are successfully funded in this competition.

More details about the NRC-HHT available resources and capacity and potential contribution to the proposals can be found in the “NRC-HHT applicant partnership fiche“. To prevent any potential conflicts of interest or privileged information sharing, the NRC-HHT will not provide any information beyond the information in the “NRC-HHT applicant partnership fiche”, and applicants must refrain from contacting the NRC-HHT at the Letter of Intent stage.

Applicants successful at the Letter of Intent stage will be provided with the NRC-HHT contact information for this program. The NRC-HHT is committed to contribute in a fair and open way to all Full Applications. As such, at the Full Application stage, applicants will be required to include the NRC-HHT as an applicant partner and to work together to prepare their application. (See Review Process and EvaluationHow to Apply and Sponsor Description sections for further details).

Competition Letter of Intent (202309LBR) Full Application (202402BRD)
Application Deadline 2023-09-20 2024-02-13

See ResearchNet for additional information.

Funding Sources

Canadian Institute of Health Research (CIHR)

This opportunity was posted by: RGCS

Last modified: August 31, 2023