The NIH Common Fund intends to publish a prize competition announcement to solicit entries for a multi-phase challenge to facilitate in vivo delivery technologies for gene editors. The NIH BRAIN Initiative will join as a contributor with other NIH-affiliated organizations.
Advancements in the genome editing technology field have enabled scientists to manipulate genomic sequences rapidly and efficiently. As a result, existing gene editing technologies such as CRISPR-cas9, base editors, and prime editors have great potential. However, there are setbacks within existing delivery technologies, including the limited delivery of gene editors to a few selected tissues and cell types, which hinders therapeutic applications. Therefore, developing a systematic, programmable in-vivo delivery system that can target diverse cells, tissues, or organs is imperative. In addition, there is also a strategic need for non-viral delivery systems capable of crossing the blood brain barrier (BBB). The BBB blocks the uptake of many pharmaceuticals, including proteins and nucleic acids, from entering the central nervous system (CNS), impeding the development of treatments for brain-related diseases.
The NIH’s Somatic Cell Genome Editing (SCGE) program, led by the NIH Office of Strategic Coordination (Common Fund), the National Center for Advancing Translational Sciences (NCATS), and the National Institute of Neurological Disorders and Stroke (NINDS), issued a notice of intent (NOT-RM-23-008) to publish a prize competition announcement to solicit entries for the multi-phase TARGETED (Targeted Genome Editor Delivery) Challenge. The BRAIN Initiative, and the National Heart, Lung, and Blood Institute (NHLBI) are also contributing to this challenge.
The TARGETED Challenge aims to improve the current state of in vivo delivery technologies for genome editors in the following areas:
- Programmable delivery systems to deliver genome editing machinery that can target specific tissues or cell types, and
- Highly efficient non-viral delivery systems capable of crossing the BBB to deliver genome editing machinery to a substantial proportion of clinically relevant cell types in the CNS.
NIH intends to award up to $6 million, which is anticipated to be awarded across the three phases of this competition. Publication of the challenge announcement and submission are planned to open in May 2023. In addition, an informational webinar is planned for June 1, 2023, at 12 pm ET. Information about the webinar will be posted on the NIH Common Fund website. Potential applicants are encouraged to sign up for the program listserv to receive updates about the challenge.
The estimated submission deadline for Phase 1 submissions is October 5, 2023. For full details, please view the notice of intent NOT-RM-23-008.