Cell and Gene Therapies for HIV Cure: Developing a Pipeline (P01 Clinical Trial Not Allowed) | RFA AI 24 013

FAQs: NIH RFA-AI-24-013 - Cell and Gene Therapies for HIV Cure: Developing a Pipeline (P01 Clinical Trial Not Allowed)

1) What is this NIH funding opportunity?

This is an NIH funding opportunity titled "Cell and Gene Therapies for HIV Cure: Developing a Pipeline (P01 Clinical Trial Not Allowed)" (RFA-AI-24-013). It supports coordinated, multi-project research programs that advance gene- and cell-based HIV cure strategies from discovery toward robust preclinical readiness, with a clear path toward eventual clinical evaluation.

2) What is the main goal of the program?

The central goal is to develop, characterize, and advance interventions intended to achieve long-term HIV remission or complete elimination of the virus. The program is designed to move promising approaches through a coherent pipeline from discovery into optimization and rigorous preclinical proof-of-concept.

3) Are clinical trials allowed under this opportunity?

No. Clinical trials are not allowed under this announcement (P01 Clinical Trial Not Allowed). The supported work is expected to be preclinical, although it should be developed with practical considerations that support later clinical evaluation under future funding or partnerships.

4) What kinds of research does NIH expect to support?

The opportunity anticipates a mix of basic science discovery and preclinical research. Example areas include foundational biology related to HIV persistence or immune mechanisms relevant to cure, technology development such as gene-editing tools, vectors, or cellular products, and translational preclinical testing including test-of-concept studies in animal models.

5) What does NIH mean by developing a "pipeline" in this context?

"Pipeline" signals an end-to-end, connected progression of work. Reviewers will likely look for discovery outputs (ideas, targets, tools) that feed into optimization, safety and performance characterization, and then into rigorous preclinical proof-of-concept packages that set the stage for later clinical entry.

6) What funding mechanism is being used, and what does it imply for applicants?

This opportunity uses the NIH P01 mechanism, which supports larger, integrated programs made up of multiple related projects sharing an overall scientific theme. A P01 application typically combines complementary projects that are structured to achieve goals that would be difficult to accomplish through separate, independent grants.

7) What would a typical P01 program look like for this NOFO?

Based on the description provided, a typical program would include multiple complementary projects spanning areas like mechanistic HIV cure science, engineering/product development for gene and cell therapies, and translational preclinical testing. The application is expected to be coordinated and integrated around a shared theme, with components that work together as a unified program.

8) Is private sector participation required?

Yes. A defining requirement is the inclusion of one or more private sector partners as active participants in the overall program.

9) What qualifies as an acceptable private sector partner role?

The private sector partner(s) are expected to contribute substantively, not just provide letters of support. The partnership should be described in a way that shows how it will accelerate translation, standardization, and readiness for later-stage development, particularly around manufacturability, platform development, quality systems, regulatory planning, scalability, and commercialization pathways.

10) Why is NIH requiring private sector partners for this opportunity?

The requirement aligns with the program's emphasis on eventual clinical translation and real-world deliverability. Industry partners often bring practical expertise in scaling processes, product characterization, quality systems, and planning for later regulatory and commercialization needs, all of which can reduce bottlenecks when moving cell and gene therapies toward clinical evaluation.

11) What practical translation issues does NIH want programs to address?

The program description highlights practical issues that can slow translation, including reproducible product characterization, dose and potency metrics, delivery approaches, and the feasibility of scaling processes for gene and cell therapy products.

12) Can the program include animal model studies?

Yes. The opportunity explicitly anticipates preclinical research, including test-of-concept studies in animal models.

13) Who is eligible to apply as the lead applicant organization?

The eligible applicant pool is broad and includes many types of U.S.-based organizations and government entities. Examples listed include state, county, and city or township 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 that are not federally recognized; public housing authorities and Indian housing authorities; nonprofits with and without 501(c)(3) status (excluding institutions of higher education where relevant); for-profit organizations other than small businesses; and small businesses, along with other categories NIH may allow.

14) Are specific institution types encouraged or highlighted as eligible?

Yes. The NOFO highlights eligibility for organizations such as Historically Black Colleges and Universities (HBCUs), Hispanic-serving institutions, Tribally Controlled Colleges and Universities (TCCUs), Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), faith-based or community-based organizations, and U.S. territories or possessions.

15) Can a foreign (non-U.S.) organization apply as the primary applicant?

No. Non-domestic (non-U.S.) entities are not eligible to apply as the primary applicant organization. The lead applicant must be a domestic eligible organization.

16) Can the project include international elements or foreign components?

Yes. While foreign organizations cannot be the primary applicant, non-domestic components of U.S. organizations are eligible to be included, and foreign components (as NIH defines them in the NIH Grants Policy Statement) are allowed when appropriately justified and structured under NIH rules.

17) What is the funding category and administrative classification provided?

The opportunity is described as a discretionary NIH grant opportunity in the health category and is associated with CFDA numbers 93.233, 93.242, 93.837, 93.838, 93.839, 93.840, and 93.855.

18) What are the key dates mentioned?

The NOFO was created on April 8, 2024. The original closing date listed is July 30, 2024.

19) Does the excerpt provide the award ceiling, expected number of awards, or budget limits?

No. The provided excerpt does not specify an award ceiling or the expected number of awards. Applicants would typically consult the full NOFO text for budget limits, project period expectations, required program structure (including any P01-specific components), and special review criteria.

20) What does NIH appear to value most in applications based on this description?

The description emphasizes integrated, multidisciplinary program execution: mechanistic HIV cure science, engineering and product development for gene and cell therapies, and rigorous preclinical evaluation that de-risks the approach for future clinical testing. It also emphasizes practical translation factors such as scalability and deliverability, and meaningful private sector participation.