NEI Update for The Ohio State University: Vision Research and Translational Opportunities

National Eye Institute (NEI) Update for The Ohio State University 14 November 2024 Tony D. Gover, Ph.D. Program Director, Division of Extramural Science Programs

NEI Mission The mission of the National Eye Institute is to eliminate vision loss and improve quality of life through vision research. To achieve this mission, NEI provides leadership to: Drive innovative research to understand the eye and visual system, prevent and treat vision diseases, and expand opportunities for people who are blind or have low vision Foster collaboration in vision research and clinical care to develop new ideas and share knowledge across other fields Recruit, inspire, and train a talented and diverse new generation of individuals to expand and strengthen the vision workforce Educate health care providers, scientists, policymakers, and the public about advances in vision research and their impact on health and quality of life Tony.gover@nih.gov 2

NEI Translational Research Opportunities Tony.gover@nih.gov 3

NOFOs Supporting Regulated Medical Product Development Research Project Grants R21,R15,R00,R01 SBIR &STTR R41,R42,R43,R44 Minimal risk trials only Bioengineering Partnerships with Industry U01 Bioengineering Research Grants R01 Blueprint MedTech UG3/UH3, U44 Blueprint Neurotherapeutics Network UG3/UH3, U44 NEI Translational Research Program R61/R33 NEI Clinical Research Study Planning Grant R34 NEI SBIR Early-Stage Clinical Trials U44 NEI Early-Stage Clinical Trials U01 NEI Collaborative Clinical Vision Research UG1 Discovery Phase I Phase II Phase III File to FDA Pre-clinical Regulatory Milestone: TRL: 1 2 3 File NDA/BLA/PMA Approval IND/IDE 4 5 6 7 8 9 510K 4

NEI Translational Research Program for Therapeutics The purpose of TRP is to encourage investigators to pursue translational activities to advance the development of biological, pharmacological, medical device, and/or combination product therapeutics for diseases and disorders of the visual systems Translational Research Program for Therapeutics (PAR-23-200: R61/R33-Clinical Trial Not Allowed) and (PAR-23-205: R33-Clinical Trial Not Allowed) R61: Exploratory phase will support research that has demonstrated significant preliminary data but has not advanced to the level of clinical translation. Budgets are limited to $500,000 in direct costs per year R33: Developmental phase focused on advancing a single therapeutic candidate through IND/IDE - enabling studies, filing an IND/IDE package with the FDA, and designing future clinical trials. Budgets are limited to $1,000,000 in direct costs per year Due Date: 16 February 2025 Tony.gover@nih.gov 5

NIH Blueprint for Neuroscience Research Milestone Driven Mechanisms: to encourage investigators (UG3/UH3 mechanism) and Small Business Concerns (SBCs) (U44 mechanism) to pursue translational activities and clinical feasibility studies to advance the development of therapeutics and diagnostics for disorders that affect the nervous or neuromuscular systems Blueprint MedTech: goal is to accelerate patient access to groundbreaking, safe, and effective medical devices Reissuance of the Translator UG3/UH3 (PAR-21-315) and U44 (PAR- 21-282) concepts were approved during May 2024 council Blueprint Neurotherapeutics Network (BPN) Program for Small Molecules (PAR-25-051 & PAR-24-063): goal is to provide funding for small molecule drug discovery and development Due Dates: 15 January 2025, 15 July 2025, 15 January 2026 Blueprint Neurotherapeutics Network for Biologics (BPN- Biologics; PAR-24-293 & PAR-24-294): goal is to provide support for biologic-based therapeutic discovery and development Due Dates: 27 January 2025, 15 July 2025, 15 January 2026 https://neuroscienceblueprint.nih.gov/ Tony.gover@nih.gov 6

Bioengineering Research Bioengineering Partnerships with Industry (BPI, PAR-24-325: U01-Clinical Trial Optional) to support milestone-driven, targeted technological development projects through strategic alliances and partnerships between academia and industry Clear description of both Industrial and academic partners involvement Leadership plan that involves both industrial and academic partners Due Dates: 28 January 2025, 28 May 2025, 27 January 2026, 27 May 2026 Bioengineering Research Grants (BRG, PAR-22-242: R01-Clinical Trial Not Allowed) to support development of innovative technologies, methods, tools, models or designs that have the potential for significant impact on biomedical research by infusing principles and concepts from quantitative sciences Apply a multidisciplinary bioengineering approach to the solution of a biomedical problem Integrate, optimize, validate, translate or otherwise accelerate the adoption of promising tools, methods and techniques for a specific research or clinical problem Due Dates: 5 February 2025, 5 June 2025 Tony.gover@nih.gov 7

Small Business Research Program (1 of 3) SBIR and STTR grant funding opportunities offer small business entrepreneurs a chance to obtain non-dilutive funding for early-stage research and development Omnibus Solicitations: Researcher-initiated ideas are proposed via the SBIR and STTR Omnibus grant solicitations Small Business Innovation Research (SBIR): PA-24-246, PA-24-245 to support small business-led projects that have high commercialization potential (Phase I, Fast-Track, and Direct Phase II) Small Business Technology Transfer (STTR): PA-24-277, PA-24-248 to support commercialization-oriented projects with US research/non-profit partnership (Phase I and Fast- Track) Phase I: Feasibility demonstrating project that may not exceed 1 year with budget $314,363 Phase II: Full R&D project after completion of Phase I, may not exceed 2 years with budget $2,095,748 Due Dates: 5 January 2025, 5 April 2025 Tony.gover@nih.gov 8

Small Business Research Program (2 of 3) NEI Small Business Innovation Research (SBIR) Cooperative Agreement for Early-Stage Clinical Trials with Greater than Minimal Risk (PAR-24-066: U44-Clinical Trial Required) Purpose: to support milestone-driven, early-stage commercialization-oriented clinical trials with greater than minimal risk and typically are Phase I or II trials Phase II: Permits budget requests up to $1,000,000 (total costs) per year, but the total costs for the entire project period should not exceed $2,000,000. Durations up to three years maximum Due Dates: 24 January 2025, 28 May 2025, 23 September 2025 Tony.gover@nih.gov 9

Small Business Research Program (3 of 3) Small Business Transition Grant for New Entrepreneurs (PAR-24-133; PAR-24-134; PAR-24- 131; PAR-24-132) Purpose: to foster the advancement and accelerate the growth of early-career scientists transitioning to entrepreneurship by simultaneously supporting their entrepreneurial development and the conduct of research and development under their direction. Mentorship: Applications must identify at least one mentor who will facilitate the successful completion of the project, from both the technical and commercial points of view. Mentor is required to commit at least 5% time Entrepreneurship Training: A career development plan for the PD/PI is required, and may include a combination of coursework, workshops, or other programs Phase I: Feasibility demonstrating project, may not exceed 1 year with a budget $306,872 (total costs) https://seed.nih.gov/ Due Dates: 5 January 2025, 5 April 2025, 5 September 2025 Tony.gover@nih.gov 10

NEI Clinical Trial Planning Grants NEI Regenerative Medicine Clinical Trial Planning Grant (R34 - Clinical Trials Not Allowed) PAR-22-135: NOFO provides support for activities central to the refinement and completion of a study protocol and procedures necessary for implementing a clinical trial to evaluate interventions (or new treatments) that restore vision in humans through regeneration of cells in the eye and visual system. Applicants may use this grant to support the development of a detailed Manual of Procedures; conduct non-clinical (non-human) work to collect safety/toxicity data; and/or conduct preliminary studies to evaluate feasibility and acceptability of regenerative strategies. NEI permits direct costs up to $150,000 per year for a maximum period is two years Due Date: 16 February 2025 NEI Clinical Research Study Planning Grant Program (R34 Clinical Trial Not Allowed) PAR-22-128: NOFO is designed to (1) permit early peer review of the rationale for the proposed clinical trial or epidemiology study, (2) provide support for the development of a complete study protocol and associated documents, including a detailed MOP, (3) support the development of other essential elements of the clinical study. NEI permits direct costs up to $150,000 per year for a maximum period is two years Due Date: 16 February 2025 Tony.gover@nih.gov 11

Eye Product Assistance Center Concept Clearance approved by NEI National Advisory Eye Council 11 October 2024 Objective: Provide direct preclinical consultation support to investigators to increase the number and quality of applications and subsequent product development efforts. Scope: The contractor will provide direct preclinical consultation support to up to 30 investigators per year to increase the number and quality of applications and subsequent product development efforts. Investigators may request up to 20 hours of consultant support for, but not limited to, the following: Regulatory Chemistry, Manufacturing, and Controls (CMC) Identification of appropriate contract research organizations (CROs) and consultants Intellectual property Commercialization Strategic partnership issues Contractor support may not be used to assist in the development, writing, or editing of research applications to be submitted to the federal government Tony.gover@nih.gov 12

For more information on NOFOs Supporting Regulated Medical Product Development Small Business Research Program NEI Translational Research Program for Therapeutics NEI Clinical Trial Planning Grants NIH Blueprint for Neuroscience Research Eye Product Assistance Center Concept Clearance Bioengineering Research Tony.gover@nih.gov 13

Questions? Tony D. Gover, Ph.D. Program Director, Division of Extramural Science Programs Tony.gover@nih.gov