
What You Should Know:
- 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 $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. 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.
Targeted Challenge Areas
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.
Phase one begins May 15th and will run for several months. 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.