Targeted Genome Editor Delivery (TARGETED) Challenge

The Targeted Genome Editor Delivery (TARGETED) Challenge is a scientific and technology demonstration competition spearheaded by the National Institutes of Health (NIH) to catalyze advancements in genome editor delivery technologies. With total cash prizes of $6 million, the Challenge is part of the NIH Common Fund’s Somatic Cell Genome Editing (SCGE) program, which aims to improve the efficacy and specificity of gene editing tools, and to accelerate their translation into clinical settings. The initiative is jointly supported by the NIH Office of the Director, the National Center for Advancing Translational Sciences (NCATS), the National Institute of Neurological Disorders and Stroke (NINDS), the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative, and the National Heart, Lung, and Blood Institute (NHLBI). Administration of the Challenge is contracted to Freelancer.com through NASA’s Center of Excellence for Collaborative Innovation. The Challenge targets two critical areas in gene therapy delivery: (1) Programmable Delivery Systems capable of delivering genome editing machinery to at least three distinct cell types, tissues, or organs, and (2) Non-viral Delivery across the Blood-Brain Barrier (BBB), where existing delivery systems fall short. Participants are tasked with developing innovative solutions that address these delivery bottlenecks and demonstrate safety, programmability, scalability, and efficiency. The end goal is to identify delivery platforms that could enable widespread clinical application of genome editing for a variety of diseases, including those impacting difficult-to-access tissues or the brain. The competition unfolds over three phases. Phase 1 focused on proposal submissions, where up to ten winning concepts were awarded $75,000 each, with additional prizes for meritorious entries. In Phase 2, participants submitted preliminary data and methodologies to demonstrate feasibility and effectiveness, with winners receiving $250,000 each. Only winners from Phase 2 could advance to Phase 3, which is divided into two sub-phases: Phase 3a and Phase 3b. Phase 3a involved demonstrating readiness for large animal testing, with awards of $50,000 (plus $40,000 for Target Area 2 participants). Phase 3b is the final stage, where submitted reagents and protocols undergo independent NIH-supported testing. Top-performing solutions in each Target Area will receive up to $625,000 for first place and $225,000 for second place. Applicants must be U.S.-based entities or teams led by U.S. citizens or permanent residents. Federal employees, judges, and those with conflicts of interest are excluded. Participants may not use federal contract funds for submission development, but may participate under specific federal grant or cooperative agreement conditions. All submissions must comply with NIH guidelines, including animal welfare laws. Submissions also require a Public Access and Dissemination Plan (PADP) to ensure broad public benefit from winning technologies, emphasizing NIH's objective to maximize accessibility and impact across diverse populations, regardless of disease prevalence. Evaluation criteria differ by target area but focus heavily on delivery efficiency, programmability, tissue specificity, and clinical translation potential. For Target Area 1, successful solutions must demonstrate delivery and editing across three or more tissue types and justify their mechanistic programmability. For Target Area 2, solutions must be non-viral and capable of traversing the BBB to deliver genome editors to relevant brain cells. Additional judging factors include market potential, scalability, and overall feasibility for clinical application. Phase 3 officially launched on March 6, 2025. Phase 3a submissions were due by July 14, 2025, and winners were announced on August 29, 2025, coinciding with the launch of Phase 3b. Participants entering Phase 3b must submit reagents and all required documentation by May 4, 2026. Final evaluations and independent validation will take place through August 2027. Phase 3 winners are expected to be announced in September 2027. The timeline requires detailed coordination with NIH-supported testing centers, with specific protocols, reagents, and shipping logistics outlined for compliance. The competition is structured to ensure rigorous evaluation while encouraging scalable, safe, and effective genome editing delivery technologies for future medical applications.