New Technologies for the In vivo Delivery of Gene Therapeutics for an HIV Cure (R01 Clinical Trial Not Allowed) | RFA AI 20 076

Opportunity Information: Apply for RFA AI 20 076

The grant opportunity titled "New Technologies for the In vivo Delivery of Gene Therapeutics for an HIV Cure (R01 Clinical Trial Not Allowed)" (Funding Opportunity Number RFA-AI-20-076) is a National Institutes of Health (NIH) research grant aimed at accelerating HIV cure-focused gene therapy by solving one of the biggest bottlenecks in the field: getting gene-based anti-HIV therapeutics safely and efficiently into the right cells inside the body. The FOA centers on the development and validation of genuinely new or significantly improved in vivo delivery strategies that can target specific cell types relevant to HIV persistence and rebound. In practical terms, the program is looking for research that moves beyond conceptual delivery ideas and instead produces evidence-backed delivery technologies that can reach intended tissues, transduce or modify the intended target cells, and do so with performance characteristics that make the approach plausible for future clinical translation.

The scientific focus is on "anti-HIV gene therapies" and, more specifically, on how to deliver them in vivo to "specific target cells." That language signals interest in technologies that can home to, enter, and function within HIV-relevant cellular reservoirs and immune compartments, rather than broadly distributing genetic payloads throughout the body. The FOA is not framed as a request for clinical testing; it is explicitly an "R01 Clinical Trial Not Allowed," meaning the funded work is expected to be preclinical or otherwise non-clinical in nature. Applicants would typically be expected to generate rigorous experimental data demonstrating delivery efficiency, target specificity, durability of expression or editing, and basic safety-related readouts appropriate to an R01 research program, without crossing into actual clinical trial activities.

Mechanistically, the announcement supports research that creates and validates innovative delivery systems for gene therapeutics. While the FOA text provided is brief, "in vivo delivery" in this context commonly encompasses approaches such as engineered viral vectors, non-viral nanoparticles, lipid-based systems, extracellular vesicle approaches, cell-targeting ligands, novel tropism/redirection strategies, and methods that improve tissue penetration and cell-type selectivity. "Validation" also implies that proposals should include well-designed studies showing that the delivery method works as intended in relevant models, with clear metrics for delivery to target cells and functional activity of the anti-HIV genetic payload once delivered. The overarching purpose is to make gene therapy strategies for HIV cure more realistic by addressing the delivery challenge that often limits efficacy, scalability, and safety.

From an administrative standpoint, this is a discretionary grant under the NIH, categorized under Health, and uses the R01 funding mechanism (a standard NIH investigator-initiated research project grant format). The CFDA numbers listed are 93.242 and 93.855, reflecting NIH program areas associated with infectious diseases and related biomedical research. The opportunity was created on 2020-10-20, and the original closing date was 2023-03-17. The source data does not specify an award ceiling or the expected number of awards, which usually means applicants would need to consult the full FOA text and NIH budget guidelines to understand likely budget ranges, project periods, and institute-specific constraints.

Eligibility is broad and includes many types of domestic U.S. organizations, such as state, county, and city 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; public housing authorities/Indian housing authorities; nonprofit organizations (both 501(c)(3) and non-501(c)(3), excluding institutions of higher education where applicable); for-profit organizations (other than small businesses); small businesses; and other eligible entities. The FOA also explicitly highlights additional eligible applicant categories, including Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Tribally Controlled Colleges and Universities (TCCUs); eligible federal government agencies; faith-based or community-based organizations; Indian/Native American Tribal Governments other than federally recognized entities; U.S. territories or possessions; regional organizations; and non-domestic (non-U.S.) entities, meaning foreign organizations can apply as well. That breadth suggests NIH is encouraging a wide range of research teams and institutional contexts to contribute engineering, virology, immunology, and translational delivery expertise to the HIV cure space.

Overall, the opportunity is best understood as an R01-level push to generate the next wave of delivery technologies that can make HIV gene therapeutics more precise, efficient, and feasible in living systems. The emphasis is on innovation in delivery coupled with strong validation, positioned upstream of human trials but intended to lay the groundwork for future clinical development by proving that the delivery problem can be overcome for the cell types and anatomical sites that matter most in HIV persistence.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "New Technologies for the In vivo Delivery of Gene Therapeutics for an HIV Cure (R01 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.242, 93.855.
• This funding opportunity was created on 2020-10-20.
• Applicants must submit their applications by 2023-03-17. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• 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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