operating grant: bringing rare disease gene therapies to clinical trial readiness

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.