The new $6M competition aims to encourage genome editing technology development and improved editing efficiency

The United States National Institutes of Health (NIH) today announced the launch of the TARGETED (Targeted Genome Editor Delivery) Challenge.

The multi-phase challenge will award up to US$6M in prize money to support NIH’s Somatic Cell Genome Editing (SCGE) commitment to developing targeted delivery systems to deliver genome editors to somatic (non-reproductive) cells of the body.

Somatic cell genome editing holds great promise in treating various diseases. However, current techniques in genome editing approaches, such as those based on CRISPR-Cas9, pose many obstacles that need to be overcome before they can be widely used in the clinic.

“To realize the promise of genome editing for treating many genetic diseases, we need genome editing technologies that can be delivered to relevant tissues and cells in a targeted way,” said Dr. Joni L. Rutter, Ph.D., Director of the National Center for Advancing Translational Sciences, which co-manages the SCGE program. “The TARGETED Challenge will help improve these technologies and speed their translation from the lab into the clinic.”

The first phase of the TARGETED Challenge is open to all eligible competitors to submit novel concepts through a written proposal. Competitors will submit proposals describing their technology, how it addresses the problem, and how they will complete the work required of the solution.

The second phase will involve the submission of preliminary results from experimental testing to show the ability to solve the challenge, while the final phase will require competitors to submit reagents and delivery protocols for independent large animal testing and validation of their solution. The Challenge will be open to new participants throughout phases one and two.

The most promising solutions will be independently tested and validated and awarded the final prizes. All solutions submitted to this contest must solve one of the two Target Areas:

Target Area 1: 

Programmable Delivery System for Gene Editing Solutions to Target Area 1 should be a highly efficient and programmable delivery system to deliver genome editing machinery that can target specific tissues or cell types. Solutions must have at least 3 configurations and be at least as efficient as existing state-of-the-art technologies. An optimal solution would be straightforward to manufacture, low-cost, scalable and have a reasonable safety profile. Technologies must have a clear relationship between what is done to modify the technology to alter specificity and how this relates to the underlying biology and/or biochemistry of the target system.

Target Area 2: 

Crossing the Blood Brain Barrier Despite major wins in genomic editing systems, crossing the blood brain barrier (BBB) remains a challenge. The BBB is a structural and functional barrier to microorganisms intended to keep harmful organisms such as fungi, bacteria, viruses, or parasites away from the brain. Solutions to Target Area 2 should be a highly efficient non-viral delivery system capable of crossing the BBB to deliver genome editing machinery to a substantial proportion of clinically relevant cell types in the brain. Solutions that can target specific cells in the CNS, but not the majority of the cells in the CNS may use that configuration as part of their solution for Target Area 2.

The TARGETED Challenge welcomes qualified entrants residing in the United States or international participants who are part of a team based in the US. Through this competition, NIH hopes to attract individuals in the genome editing field as well as technology developers outside of the field, including drug delivery technology developers, nanotechnology experts or biomedical engineers.

Phase one begins May 15th and will run for several months.

The TARGETED Challenge is part of the Somatic Cell Genome Editing (SCGE) Program funded by the NIH Common Fund and co-managed by the National Center for Advancing Translational Sciences (NCATS) and 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) are also contributors to this Challenge. NIH has contracted with Freelancer.com to support the design, implementation and management of the challenge through a multi-award contract from the NASA Tournament Lab. For more information on the contest, please visit: https://www.freelancer.com/nih/targeted-challenge.

About the NIH SCGE Program

The NIH Common Fund’s Somatic Cell Genome Editing (SCGE) program is working to improve the efficacy and specificity of gene editing approaches to help reduce the burden of common and rare diseases caused by genetic changes. SCGE is developing quality tools to perform and assess effective and safe genome editing in non-reproductive (“somatic”) cells of the body. These research tools will be made widely available to the research community to reduce the time and cost required to develop new therapies.

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This page last reviewed on May 15, 2023.