Notice of Upcoming Funding Opportunity: Operating Grants: Bringing Rare Disease Gene Therapies to Clinical Trial Readiness

This funding opportunity is now available on ResearchNet .

CIHR is pleased to provide information on an upcoming funding opportunity for the Operating Grants: Bringing Rare Disease Gene Therapies to Clinical Trial Readiness. This funding is a part of the National Strategy for Drugs for Rare Diseases and is supported through Federal Budget 2019.

CIHR will be hosting a webinar to inform participants about the requirements of this funding opportunity and to answer questions. To find out more information, visit the Webinars page.


Rare diseases 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 globallyFootnote 1.

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 rodent models of disease to human clinical trial readiness. This funding opportunity seeks to fund research to bridge this gap.


The specific objectives of the funding opportunity are to:

  • Increase development of gene therapies for rare disease clinical trials in Canada;
  • Generate the evidence required for first-in-human clinical trials, in part by working with Canada’s biomanufacturing capacity and health technology regulator; and,
  • Increase capacity within Canada to improve readiness of gene therapies for first-in-human clinical trials.

Funds Available

Up to $2,275,000 is available to fund two grants for four years, for a total of $1,137,500 per grant.

Anticipated timeline

These dates are estimates and subject to change.

Program Launch: Summer 2023
Letter of Intent Deadline: Early Fall 2023
Letter of Intent Notice of Decision: Fall 2023
Full Application Deadline: Winter 2024
Full Application Notice of Decision: Summer 2024
Funding Start Date: April 2024

Contact Information

For general inquiries please contact:

CIHR Contact Centre
Telephone: 613-954-1968
Toll Free: 1-888-603-4178


The information contained herein is anticipatory only and does not represent an official funding commitment by the Canadian Institutes of Health Research. Accordingly, it may differ from the official funding opportunity that will be published on ResearchNet.

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