NEI Audacious Goals Initiative: Translation-Enabling Models to Evaluate Survival and Integration of Regenerated Neurons in the Visual System (U24 Clinical Trials Not Allowed) | RFA EY 17 003

Opportunity Information: Apply for RFA EY 17 003

The National Eye Institute (NIH) is seeking proposals under its Audacious Goals Initiative to build better, more clinically relevant experimental models for testing whether regenerated retinal neurons can actually survive, connect properly, and restore visual function. This funding opportunity is focused on translation-enabling models, meaning model systems designed to bridge the gap between early laboratory work and eventual human therapies. The central problem it aims to address is that many existing animal and lab models do not closely match human visual anatomy, retinal cell organization, or disease features, which makes it hard to predict whether cell-replacement strategies for blindness will truly work in people.

The scientific target is regeneration and functional integration of two key retinal neuron types: photoreceptors (PRCs), which capture light and begin visual signaling, and retinal ganglion cells (RGCs), which transmit visual information from the retina to the brain through the optic nerve. Applicants are expected to create or refine models that mimic critical aspects of human blinding diseases that could plausibly be treated through regenerative approaches. These models can be generated in a range of ways, including transgenic gene insertion or deletion, gene editing methods, chemically or physically induced damage, or other strategies that recreate disease-relevant defects or create controlled lesions suitable for testing cell replacement. While multiple model types are allowed, the FOA strongly encourages the use of non-human primates or other cone-dominant species because they more closely resemble the human retina in structure and function (including features relevant to central vision).

A defining expectation of the program is that teams do more than just develop a disease-like model. They are also expected to attempt treatment within that model using an approach that regenerates PRCs and/or RGCs and supports formation of appropriate neural connections. In practice, this means applicants should pair their model with a clear regeneration or cell-replacement strategy, such as transplantation of adult stem cells, neural precursors, stem cell-derived progenitor cells, or in situ reprogramming approaches (for example, converting intrinsic retinal cells like glia into neurons). The emphasis is on demonstrating not only cell survival, but meaningful integration into existing retinal circuitry, since integration is what ultimately determines whether vision can be restored.

Because this initiative is meant to accelerate translation, the FOA places heavy weight on quantitative, objective readouts of success. Awardees are expected to use rigorous measures to assess survival and functional integration of regenerated cells, potentially including electrophysiology (to show neurons are firing and responding appropriately), functional imaging, behavioral assessments of vision, or other technologies that can convincingly demonstrate circuit-level integration and improvement in visual function. In other words, the program is not satisfied with anatomical presence alone; it wants evidence that regenerated neurons are doing the job they are supposed to do within the visual system.

From an administrative standpoint, this is a discretionary NIH opportunity run as a cooperative agreement (U24), which typically means NIH staff will have substantial involvement or partnership in the funded work compared with a standard research grant. The FOA is explicitly "Clinical Trials Not Allowed," so the work must remain preclinical and focused on model and tool development rather than testing interventions in human participants. The opportunity is identified as RFA-EY-17-003 under CFDA 93.867, with an award ceiling of up to $1,000,000. The original closing date listed for the competition was March 21, 2018, indicating this is a specific-time solicitation rather than a permanently open mechanism.

Eligibility is broad and includes many types of U.S. organizations and governments, such as public and private institutions of higher education, nonprofits (with and without 501(c)(3) status), for-profit organizations (including small businesses), and various levels of government (state, county, city/township, special districts), as well as tribal governments and tribal organizations. The FOA also highlights inclusion of entities such as Historically Black Colleges and Universities (HBCUs), Hispanic-serving institutions, Tribally Controlled Colleges and Universities (TCCUs), Alaska Native and Native Hawaiian Serving Institutions, and Asian American Native American Pacific Islander Serving Institutions (AANAPISIs). In addition, non-U.S. (foreign) organizations, regional organizations, faith-based or community-based organizations, and U.S. territories or possessions are listed among other eligible applicant categories.

Overall, the opportunity is designed to produce shared, enabling resources for the vision research community: models, tools, devices, and related platforms that make it more realistic to evaluate whether regenerative therapies for retinal diseases can succeed. The core deliverable is a model system that more faithfully reflects human retinal disease and can be used to test, with strong quantitative evidence, whether regenerated photoreceptors and/or retinal ganglion cells survive, integrate into neural circuits, and measurably restore visual function.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "NEI Audacious Goals Initiative: Translation-Enabling Models to Evaluate Survival and Integration of Regenerated Neurons in the Visual System (U24 Clinical Trials Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.867.
• This funding opportunity was created on 2017-12-07.
• Applicants must submit their applications by 2018-03-21. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Each selected applicant is eligible to receive up to $1,000,000.00 in funding.
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

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