Targeted Genome Editor Delivery (TARGETED) Challenge

The National Institutes of Health (NIH) is launching the Targeted Genome Editor Delivery (TARGETED) Challenge to improve the delivery methods for gene-editing technologies. Recent developments in gene-editing have allowed scientists to quickly and efficiently modify genomic sequences. Still, the current forms of delivery don't consistently reach many target tissues and cell types in sufficient quantities, which prevents these technologies from more widespread clinical application.

The TARGETED Challenge comes with a prize amount of $6,000,000, split across a three-phase competition. As part of this challenge, the NIH seeks participants with innovative ideas or early-stage solutions that can enhance the delivery of gene-editing technologies. Participants can win up to $1,000,000 and have their solution tested and validated in large animal models, with the NIH overseeing independent evaluations.

The Challenge's primary target areas are developing a Programmable Delivery System for Gene Editing and finding a way to cross the Blood-Brain Barrier with the technology. Participants can submit solutions for each target area, but they must be separate proposals. Of these submissions, up to ten that best fit the requirements will be awarded up to $75,000. An additional $50,000 may be given based on how the proposed solution meets the judging criteria.

The second phase of the competition requires participants to submit data demonstrating their solution's delivery and editing performance. Although participation in phase one isn't mandatory for phase two, it is strongly encouraged. The top ten winners of the second phase will each receive $250,000 and are qualified to participate in Phase 3.

Phase 3, consisting of stage 3a and 3b, requires participants to demonstrate their technology is ready for large animal testing. Up to 6 participants will receive $50,000 and will progress to develop protocols for further testing. The best performing solutions in each target area will be awarded cash prizes, with potential earnings up to $1,000,000 in total for those who successfully participate in all three stages.

The TARGETED Challenge is overseen by the NIH Common Fund's Somatic Cell Genome Editing (SCGE) program, with the National Center for Advancing Translational Sciences (NCATS) and the National Institute of Neurological Disorders and Stroke (NINDS) providing leadership. Other contributors include the Brain Research Through Advancing Innovative Neurotechnologies (BRAIN) Initiative and the National Heart, Lung, and Blood Institute (NHLBI). The Challenge is administered with support from Freelancer.com under a NASA contract on behalf of the NIH.

This Challenge aims to address the difficulties in delivering genome editors to disease-relevant cell types in the various organs associated with different diseases. Currently, even when successful delivery is achieved, the efficiency rate — or the percentage of cells that ultimately get edited — is often lower than ideal. The TARGETED Challenge is a continuation of the SCGE program's efforts to improve and accelerate the clinical development of genome editing to reduce the burden of common and rare diseases caused by genetic mutations. The efficacy of solutions for the two strategic Target Areas will be a transformative step for genome editing technology, fulfilling its full potential in treating diseases.