Targeted Genome Editor Delivery (TARGETED) Challenge

The Targeted Genome Editor Delivery (TARGETED) Challenge is a $6,000,000 competitive initiative launched by the National Institutes of Health (NIH), through the Somatic Cell Genome Editing (SCGE) program under the NIH Common Fund. This multiyear scientific and technology demonstration challenge seeks to address persistent barriers in the field of genome editing, particularly the lack of effective in vivo delivery technologies for gene editing tools. With recent advances in tools like CRISPR-Cas9, base editors, and prime editors, the bottleneck in therapeutic development has shifted to delivery—especially to hard-to-reach tissues and across biological barriers such as the blood-brain barrier. The TARGETED Challenge is designed to fund and evaluate solutions that significantly advance delivery mechanisms, facilitating clinical translation. The program is segmented into two strategic target areas. The first area, Programmable Delivery System for Gene Editing, emphasizes the development of programmable systems capable of targeting at least three distinct tissues, cell types, or organs with high specificity and efficiency. The second area, Crossing the Blood-Brain Barrier, prioritizes non-viral delivery technologies capable of traversing the barrier and delivering genome editors to relevant cell types in the brain. Each area carries distinct requirements for safety, scalability, and innovation, but both aim to enable successful genome editing performance validated through NIH-supported independent evaluation in large animal models. The challenge is organized into three progressive phases. Phase 1 required submission of a written proposal describing the technology and its application to one of the target areas. Up to ten winners were selected to receive $75,000 each, with additional $50,000 awards granted to other meritorious proposals. In Phase 2, participants are expected to submit preliminary data demonstrating editing performance, delivery efficiency, and methodological clarity. Winners of Phase 2 will each receive $250,000 and qualify for Phase 3. Phase 3, split into subphases 3a and 3b, involves submission of comprehensive information validating readiness for large animal testing. Participants who proceed to Phase 3b will undergo NIH-funded independent validation. Top performers in each target area can win prizes of $625,000, with secondary awards of $225,000 and honorable mentions for third place. The challenge is designed not only to stimulate innovation but also to ensure broad public access to the resulting technologies. NIH mandates submission of a Public Access and Dissemination Plan (PADP) with each proposal to encourage downstream licensing and commercialization while ensuring societal benefit. The ideal solutions should be programmable, efficient, cost-effective, and scalable, with a well-characterized safety profile. Participants may submit multiple distinct proposals but can only receive one prize per solution, even if a single solution qualifies for both target areas. Partners in the challenge include the National Center for Advancing Translational Sciences (NCATS), National Institute of Neurological Disorders and Stroke (NINDS), the BRAIN Initiative, and the National Heart, Lung, and Blood Institute (NHLBI). The challenge is administered by Freelancer.com under a NASA contract on behalf of NIH. Statutory authority is granted under the COMPETES Reauthorization Act, which empowers federal agencies to use competitions to drive innovation. The timeline spans from May 2023 to August 2027. Phase 2 submissions are due by January 10, 2025, with winners announced in May 2025. Phase 3a closes on July 21, 2025, followed by reagent delivery for 3b by May 4, 2026. Final winners will be announced in August 2027. The challenge is recurring and structured to allow rolling participation across its multi-phase schedule, with future cycles likely reflecting similar timelines and submission structures.