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

Eligibility

For an application to be eligible at the LOI stage, all the requirements stated below must be met:

1. The Nominated Principal Applicant (NPA) must be one of the following:
   1. an independent researcher affiliated with a Canadian postsecondary institution and/or its affiliated institutions (including hospitals, research institutes and other non-profit organizations with a mandate for health research and/or knowledge translation) at the time of funding.
      OR
   2. an individual affiliated with an Indigenous non-governmental organization in Canada with a research and/or knowledge translation mandate;
      OR
   3. an Indigenous non-governmental organization in Canada with a research and/or knowledge translation mandate.
2. The NPA must have their substantive role in Canada for the duration of the requested grant term.
3. The Institution Paid must be authorized to administer CIHR funds before the funding can be released (see Administration of Funds).
4. The Research Team must include each of the following roles. Note that an individual can fulfill multiple roles.
   1. The research team must include an Early Career Researcher (ECR) identified as an NPA or Principal Applicant (PA).
   2. At least one project participant must be a knowledge user (including but not limited to decision makers, policy-makers, clinician scientists, health professionals) identified as Principal Knowledge User (PKU) or Knowledge User (KU).
   3. In addition to the Nominated Principal Applicant and Knowledge User, the team must include at least one patient/caregiver/family member in the role of PKU, KU or Co-Applicant as appropriate.
   4. The applicant team must have a Sex and Gender Champion and an Equity, Diversity and Inclusion (EDI) Champion as NPA, PA or Co-Applicant:
      1. Combined, the champions must have experience in (i) sex- and gender-based analysis (SGBA) or with gender diversity in the community; (ii) fostering EDI in research and/or applied settings (e.g., promoting equitable access to research participation or inclusion of typically underrepresented population groups in decision making), and (iii) creating Sex and Gender and EDI plans for diversity of team composition;
      2. These individuals may hold other roles within the team in addition to standing as the team's Sex and Gender and EDI champions;
      3. The Sex and Gender and EDI champion roles may be served by the same individual or different individuals.
   5. Any research involving Indigenous Peoples must include at least one applicant who self-identifies as Indigenous (First Nations, Inuit or Métis) and/or provides evidence of the capacity and experience to work with Indigenous Peoples in a meaningful and culturally safe way (see How to Apply for more details).

In addition to the requirements listed above, for an application to be eligible for the full application stage:

1. The NPA* and the Sex and Gender Champion, if different from the NPA, must have successfully completed one of the sex- and gender-based analysis training modules available online through the CIHR Institute of Gender and Health and submit a Certificate of Completion. Select and complete the training module most applicable to your project. Applicants are encouraged to review the page "How to integrate sex and gender in research" on the CIHR website.
   1. *Organizations as NPAs: For organizations applying as the NPA, a representative of the organization must complete the training module on the organization's behalf.
2. The NPA must provide a signed Partner Letter from the NRC-HHT, confirming that the NRC-HHT collaborated with the applicant's team to prepare their application and that the NPA will have access to the NRC-HHT resources/expertise to perform the work proposed in the application (see How to Apply section).

Summary

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 Evaluation, How to Apply and Sponsor Description sections for further details).

Objectives

The specific objectives of this funding opportunity are to:

• Increase and advance the 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 (National Research Council of Canada) and health technology regulator (Health Canada); and,
• Increase current and future capacity across the Canadian rare disease landscape (i.e., among researchers, knowledge users, patient(s)/caregiver(s)/family) to improve readiness of gene therapies for first-in-human clinical trials.