Opportunity Information: Apply for RFA NS 23 017
The NIH funding opportunity "Optimization of Genome Editing Therapeutics for Alzheimer's Disease and Alzheimer's Disease-Related Dementias (AD/ADRD) (U01 - Clinical Trials Not Allowed)" (Funding Opportunity Number RFA-NS-23-017) is a cooperative agreement designed to move genome editing therapy concepts closer to the point where they can enter formal IND-enabling development. The focus is not on discovering brand-new targets from scratch, but on taking therapeutic leads that already have strong proof-of-concept results in appropriate experimental models and doing the deeper, practical work needed to turn them into realistic drug candidates. In other words, the FOA is aimed at the optimization stage: strengthening the evidence package and refining the therapeutic approach so it is positioned for the kinds of studies required before a first-in-human program could be considered.
A central theme of the announcement is that funded projects should produce an optimized genome editing therapeutic candidate by the end of the project period. That optimized candidate is expected to demonstrate qualities that matter for translation, such as measurable bioactivity (the edit does what it is intended to do), manufacturability (the therapy can be produced in a consistent, scalable way), biodistribution (it reaches the relevant tissues and cell types, especially in the central nervous system where delivery is a major challenge), and evidence of in vivo efficacy and/or clear target engagement. Target engagement is described in a practical way, including measurement of proximal downstream effects, meaning applicants are expected to show that editing the target produces the immediate biological changes that should logically connect to therapeutic benefit. The FOA also highlights the importance of determining optimal dosing, implying a need for dose-ranging work and exposure-response relationships that can support later development decisions.
This announcement is explicitly broad across the Alzheimer's and dementia landscape rather than being restricted to a single diagnosis. It supports genome editing therapeutic development for Alzheimer's disease as well as multiple categories of Alzheimer's disease-related dementias, including frontotemporal dementia (FTD), Lewy body dementias (LBD) such as dementia with Lewy bodies (DLB) and Parkinson disease dementia (PDD), vascular contributions to cognitive impairment and dementia (VCID), and multiple etiology dementias (MED). The practical implication is that applicants can propose editing-based strategies relevant to different disease mechanisms across this spectrum, as long as the proposal is grounded in convincing proof-of-concept and focuses on optimization toward an IND-ready package rather than exploratory discovery.
The award mechanism is a U01 cooperative agreement, which typically means NIH program staff have substantial scientific and programmatic involvement compared with a standard research project grant. This structure is often used for milestone-driven, translational efforts where coordination with NIH expectations, go/no-go criteria, and deliverables is important. The FOA also specifies "Clinical Trials Not Allowed," which means the supported work is preclinical or otherwise non-clinical in nature. Applicants should interpret that as a requirement to keep the project centered on candidate optimization and preclinical development activities rather than enrolling human participants in interventional clinical studies.
From an administrative standpoint, the opportunity falls under the health funding activity category and lists CFDA numbers 93.853 and 93.866. The sponsoring agency is the National Institutes of Health. The original closing date provided is 2022-11-30, and the opportunity record shows a creation date of 2022-08-24. The stated award ceiling is $650,000, and while the expected number of awards is not clearly specified in the provided source text, applicants would typically plan budgets and scope around the ceiling and the milestone-oriented nature of a U01.
Eligibility is broad and includes many common public and private applicant types: state, county, and local governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribal organizations (including those other than federally recognized tribal governments); public housing authorities/Indian housing authorities; nonprofits with and without 501(c)(3) status (excluding institutions of higher education in those categories); for-profit organizations other than small businesses; and small businesses. In addition, the FOA calls out a wide set of "other eligible applicants" that NIH often highlights to encourage diverse institutional participation, including Alaska Native and Native Hawaiian Serving Institutions, AANAPISIs, Hispanic-serving institutions, HBCUs, tribally controlled colleges and universities (TCCUs), faith-based or community-based organizations, eligible federal agencies, regional organizations, non-U.S. entities (foreign organizations), and U.S. territories or possessions. The net effect is that the program is open to a wide range of organizations capable of doing advanced genome editing therapeutic optimization, including academic groups, nonprofits, and industry teams, potentially in collaboration.
Taken together, the FOA is best read as a translational push: it is intended for teams that already have a credible genome editing therapeutic lead relevant to AD/ADRD and now need support to refine the candidate and generate the kinds of data that de-risk development. Competitive projects under this announcement would be expected to articulate a clear optimization plan, demonstrate why the current lead is already compelling, and propose rigorous studies that establish delivery and distribution, editing activity and downstream biological impact, performance in relevant in vivo models, manufacturability considerations, and rational dosing strategies, all aligned with the end goal of positioning the candidate for IND-enabling work even though the FOA itself does not fund clinical trials.Apply for RFA NS 23 017
- The National Institutes of Health in the health sector is offering a public funding opportunity titled "Optimization of Genome Editing Therapeutics for Alzheimer's Disease and Alzheimer's Disease-Related Dementias (AD/ADRD) (U01 - 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): 93.853, 93.866.
- This funding opportunity was created on 2022-08-24.
- Applicants must submit their applications by 2022-11-30. (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 $650,000.00 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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FAQs: NIH RFA-NS-23-017 (U01) - Optimization of Genome Editing Therapeutics for AD/ADRD (Clinical Trials Not Allowed)
What is this funding opportunity?
This NIH funding opportunity is titled "Optimization of Genome Editing Therapeutics for Alzheimer's Disease and Alzheimer's Disease-Related Dementias (AD/ADRD) (U01 - Clinical Trials Not Allowed)" and has Funding Opportunity Number RFA-NS-23-017. It uses a U01 cooperative agreement mechanism to support milestone-driven, translational work aimed at advancing genome editing therapy concepts toward IND-enabling development.
What is the main goal of the FOA?
The main goal is to produce an optimized genome editing therapeutic candidate by the end of the project period, with a stronger, more practical evidence package that helps position the candidate for the kinds of studies typically required before a first-in-human program could be considered.
Is this FOA meant for discovery of new targets?
No. The focus is not on discovering brand-new targets from scratch. It is aimed at optimizing therapeutic leads that already have strong proof-of-concept results in appropriate experimental models.
What does "optimization" mean in the context of this FOA?
In this FOA, optimization refers to the deeper, practical development work needed to turn a promising genome editing therapeutic lead into a realistic drug candidate. This includes strengthening translational evidence, refining the therapeutic approach, and generating data that de-risk development.
What kinds of qualities should the optimized candidate demonstrate?
The optimized candidate is expected to demonstrate translationally relevant qualities such as measurable bioactivity, manufacturability, biodistribution (especially to relevant central nervous system tissues and cell types), and evidence of in vivo efficacy and/or clear target engagement.
What does "bioactivity" mean here?
Bioactivity is described as evidence that the edit does what it is intended to do, meaning applicants should be able to measure and demonstrate intended genome editing activity aligned with the therapeutic strategy.
What does the FOA emphasize about biodistribution and the CNS?
The FOA highlights biodistribution as a key translational requirement and notes that delivery is a major challenge in the central nervous system. Projects are expected to address whether the therapeutic reaches relevant tissues and cell types, particularly in the CNS.
What is meant by "target engagement" in this FOA?
Target engagement is framed in a practical way and includes measuring proximal downstream effects. That means applicants should show that editing the target produces immediate biological changes that logically connect to therapeutic benefit.
Does the FOA require in vivo efficacy data?
The FOA expects evidence of in vivo efficacy and/or clear target engagement. Applicants should plan studies that credibly establish performance in relevant in vivo settings and/or show strong target engagement with measurable downstream impact.
Does this FOA expect dose optimization?
Yes. The FOA highlights determining optimal dosing and implies the need for dose-ranging work and exposure-response relationships that can support later development decisions.
Which diseases and dementias are in scope?
The FOA is broad across Alzheimer's disease and Alzheimer's disease-related dementias (AD/ADRD). It includes (among others) frontotemporal dementia (FTD), Lewy body dementias (LBD) such as dementia with Lewy bodies (DLB) and Parkinson disease dementia (PDD), vascular contributions to cognitive impairment and dementia (VCID), and multiple etiology dementias (MED).
Is the FOA limited to one diagnosis or mechanism?
No. The announcement is explicitly broad across the Alzheimer's and dementia landscape rather than being restricted to a single diagnosis, as long as the proposed genome editing strategy is grounded in convincing proof-of-concept and focuses on optimization toward an IND-ready package.
What award mechanism is used, and why does it matter?
The mechanism is a U01 cooperative agreement. This typically means NIH program staff have substantial scientific and programmatic involvement compared with a standard research project grant, and the effort is often milestone-driven with defined expectations, deliverables, and potential go/no-go criteria.
Are clinical trials allowed under this FOA?
No. The FOA specifies "Clinical Trials Not Allowed," meaning the supported work must be preclinical or otherwise non-clinical in nature and should not involve enrolling human participants in interventional clinical studies.
Who is the sponsoring agency?
The sponsoring agency is the National Institutes of Health (NIH).
What CFDA numbers are associated with this opportunity?
The opportunity lists CFDA numbers 93.853 and 93.866.
What is the award ceiling?
The stated award ceiling is $650,000.
Is the expected number of awards provided?
The provided source text does not clearly specify the expected number of awards.
What are the key dates listed for this opportunity?
The opportunity record shows a creation date of 2022-08-24, and the original closing date provided is 2022-11-30.
What types of organizations are eligible to apply?
Eligibility is broad and includes state, county, and local governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribal organizations (including those other than federally recognized tribal governments); public housing authorities/Indian housing authorities; nonprofits with and without 501(c)(3) status (excluding institutions of higher education in those nonprofit categories); for-profit organizations other than small businesses; and small businesses.
Are foreign organizations or non-U.S. entities eligible?
Yes. The FOA includes non-U.S. entities (foreign organizations) among "other eligible applicants."
Are U.S. territories or possessions eligible?
Yes. U.S. territories or possessions are listed among "other eligible applicants."
Does the FOA encourage participation from specific institution types?
Yes. The FOA highlights a range of "other eligible applicants" that NIH often calls out to encourage broad participation, including Alaska Native and Native Hawaiian Serving Institutions, AANAPISIs, Hispanic-serving institutions, HBCUs, and tribally controlled colleges and universities (TCCUs), among others.
Are faith-based or community-based organizations eligible?
Yes. Faith-based or community-based organizations are included in the list of other eligible applicants.
Are federal agencies eligible applicants?
Yes. Eligible federal agencies are listed among other eligible applicants.
What kind of project is most competitive under this FOA based on the description?
Based on the description provided, a competitive project would start with a credible genome editing therapeutic lead relevant to AD/ADRD and propose a clear optimization plan with rigorous studies that address delivery and distribution (especially in the CNS), editing activity and downstream biological impact, in vivo efficacy and/or target engagement, manufacturability considerations, and rational dosing strategies, all aligned with the goal of moving the candidate closer to IND-enabling development.
Does this FOA fund IND-enabling studies directly?
The FOA is designed to move projects closer to the point where they can enter formal IND-enabling development. It emphasizes positioning and readiness for that stage, while keeping the supported work in the preclinical/non-clinical space (and not funding clinical trials).
How should applicants think about scope and budgeting?
Applicants would typically plan scope and budgeting around the $650,000 award ceiling and the milestone-oriented, cooperative agreement nature of a U01, which generally involves defined deliverables and close coordination with NIH program expectations.
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