The NEI Translational Research Program for Therapeutics (R33 Clinical Trial Not Allowed) supports the final stages of preclinical development of therapies, medical devices, and combination products for visual system diseases or disorders. Administered by the National Eye Institute under the National Institutes of Health, this funding opportunity focuses on advancing single therapeutic candidates through Investigational New Drug (IND) or Investigational Device Exemption (IDE)-enabling studies, preparing IND/IDE packages for submission to the U.S. Food and Drug Administration, and designing future clinical trials. The ultimate goal is to facilitate the transition of promising therapeutic discoveries from laboratory research to clinical application.

This program specifically targets ocular diseases, including retinal diseases, corneal diseases, cataract, glaucoma, optic neuropathies, strabismus, amblyopia, and low vision rehabilitation. Research must aim to deliver clear, milestone-driven results that demonstrate readiness for clinical translation within a three-year project period. Activities supported under the R33 phase include cGMP manufacturing of materials for IND-enabling studies, toxicology and pharmacology studies for dose extrapolation, and preparation of IND/IDE regulatory documents. Applications must include evidence of pre-IND/IDE meetings with the FDA to ensure alignment with regulatory requirements.

Eligible applicants include higher education institutions, nonprofits with or without 501(c)(3) status, for-profit organizations, and local and state governments. Foreign organizations are not eligible to apply, but foreign components of U.S. organizations are allowed. Multidisciplinary teams are strongly encouraged, comprising non-clinical and clinical scientists, regulatory experts, manufacturing specialists, and bioethics professionals. Applicants must demonstrate clear milestones that are specific, measurable, achievable, relevant, and time-bound (SMART) to facilitate progress evaluation throughout the project period.

The total project budget for the R33 phase must not exceed $1,000,000 in direct costs per year, with a maximum project period of three years. Applicants must include key documents such as a Target Product Profile summarizing therapeutic goals, FDA meeting minutes, a detailed Gantt chart with milestones, and an Intellectual Property Strategy outlining any patent filings, licensing agreements, or constraints affecting therapeutic development. Compliance with NIH’s Data Management and Sharing Policy is required, and applicants must submit a comprehensive plan detailing data management practices.

Evaluation criteria focus on the significance and innovation of the proposed research, rigor and feasibility of the approach, and the expertise and resources of the investigative team. Applications must present robust milestones with clear go/no-go decision points, demonstrate alignment with FDA regulatory requirements, and provide a well-structured plan for achieving therapeutic development goals. Projects proposing clinical trials, observational studies, or multi-site trials will be considered non-responsive and withdrawn from consideration.

Key application deadlines include the Letter of Intent due by January 16, 2025, and the full application submission deadline on February 16, 2025. Scientific merit review will occur in June 2025, with final funding decisions expected by December 2025. Applicants are encouraged to consult NIH program staff well in advance of submission to ensure alignment with program goals and requirements. Further information, submission details, and application guidelines are available through Grants.gov, NIH ASSIST, and the eRA Commons platforms.