Non-Viral Technologies for in vivo Delivery of Genome Editors (R43/R44 Clinical Trial Not Allowed) | PAR 20 098

Opportunity Information: Apply for PAR 20 098

The grant opportunity titled "Non-Viral Technologies for in vivo Delivery of Genome Editors (R43/R44 Clinical Trial Not Allowed)" (Funding Opportunity Number PAR 20-098) is a National Institutes of Health (NIH) small business funding program focused on advancing practical, non-viral delivery systems for genome editing tools inside the body. The central aim is to help small companies develop and evaluate delivery technologies that can transport genome editors to the right somatic (non-reproductive) cells and tissues in living organisms, with the long-range intention that these delivery approaches can eventually be strong enough and reliable enough to support human clinical translation for treating disease. In other words, the program is not mainly about discovering new gene targets or proving a disease mechanism; it is about solving one of the biggest bottlenecks in genome editing: getting editors to the correct cells in vivo safely, efficiently, and reproducibly without using viral vectors.

This opportunity uses the NIH SBIR/STTR-style R43/R44 grant mechanisms, which are designed specifically for small businesses and emphasize development milestones and technology maturation. These mechanisms generally support early-stage feasibility work followed by more advanced development, which fits the program's purpose of moving delivery platforms from concept and prototype toward more rigorous preclinical readiness. While the scientific direction is explicitly geared toward eventual clinical use, the funding notice makes it clear that clinical trials are not allowed under this particular announcement. That typically means applicants should plan for preclinical development activities, such as formulation optimization, biodistribution and delivery efficiency studies, dose-ranging work, safety and tolerability assessments in relevant models, manufacturability or scalability considerations, and other studies that de-risk the delivery system before any first-in-human testing would be appropriate.

The technologies of interest are non-viral, meaning the program is looking for alternatives to viral vectors (like AAV or lentivirus) for delivering genome editing components. The genome editors themselves could include different classes of editing systems (for example, nuclease-based editors or newer editing modalities), but the emphasis of the announcement is on the delivery technology rather than the editing chemistry alone. The delivery challenge in vivo is multi-layered, so strong proposals typically address key practical issues such as tissue targeting (getting the payload to disease-relevant organs or cell types), cellular uptake, endosomal escape, controlled release, stability in circulation, avoidance of unwanted immune activation, minimization of off-target distribution, and the ability to achieve therapeutic levels of editing in a repeatable way. Because the goal is eventual translation, applicants are also implicitly encouraged to consider real-world constraints like dosing route, repeat dosing potential, and how the delivery system could be produced and quality-controlled later under more stringent manufacturing expectations.

Eligibility is limited to small businesses, consistent with the SBIR/STTR program intent to stimulate commercialization-oriented R&D. The announcement also sets clear boundaries around foreign participation. Non-U.S. entities (foreign institutions) are not eligible to apply, and non-U.S. components of U.S. organizations are not eligible to apply either. However, the notice indicates that certain "foreign components" may be allowed as defined by the NIH Grants Policy Statement, which usually means that, while the applicant organization must be domestic and eligible, it may be permissible to include discrete elements of the work performed outside the U.S. if they are well-justified, clearly necessary, and compliant with NIH policy. This distinction matters for teams that want to access a unique capability, reagent, animal model, or specialized testing resource abroad, but the core applicant and primary performance site need to remain compliant with the domestic small business eligibility rules.

Administratively, this is a discretionary grant program in the health funding category, run by NIH, with CFDA number 93.350. The opportunity was created on 2020-02-06, and the original closing date listed is 2022-01-05. The listing does not provide an award ceiling or an expected number of awards in the provided data, so applicants would typically refer to the full NIH announcement text and any associated institute-specific guidance for budget expectations, project period limits, and review considerations. Overall, the program is best understood as targeted funding for small businesses trying to build non-viral in vivo delivery platforms that can make therapeutic genome editing more feasible and ultimately ready for human testing, while keeping the supported work firmly in the preclinical development space rather than clinical trial execution.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Non-Viral Technologies for in vivo Delivery of Genome Editors (R43/R44 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): 93.350.
• This funding opportunity was created on 2020-02-06.
• Applicants must submit their applications by 2022-01-05. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Eligible applicants include: Small businesses.

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