RARE Daily

National Institutes of Health Launches Challenge for Gene Editing Delivery Systems

May 17, 2023

Rare Daily Staff

The National Institutes of Health said it has launched the TARGETED (Targeted Genome Editor Delivery) Challenge, a multi-phase competition with up to $6 million in prize money to develop targeted systems to deliver genome editors to somatic cells of the body.

The challenge is intended to support NIH’s Somatic Cell Genome Editing (SCGE) commitment to developing targeted delivery systems to deliver genome editors to somatic 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 Joni Rutter, 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.

All solutions submitted to this contest must solve one of the two target areas. The first is target area focuses on a programmable delivery system for gene editing solutions that can target specific tissues or cell types. Solutions must have at least three 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.

The second target area involves delivering gene editors across the blood brain barrier. The blood brain barrier 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 should be a highly efficient non-viral delivery system capable of crossing the blood brain barrier to deliver genome editing machinery to a substantial proportion of clinically relevant cell types in the brain.

Photo: Joni Rutter, director of the National Center for Advancing Translational Sciences

The most promising solutions will be independently tested and validated and awarded the final prizes.

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