March 15, 2023
Anticipated Publication Date of Challenge Announcement: May 15, 2023
Anticipated (Registration) Submission Start Date: May 15, 2023
Anticipated (Registration closes) Submission Due Date: October 5, 2023
Anticipated Award Date: December 15, 2023 Anticipated Start Date: TBD
NOT-RM-22-013 Request for Information (RFI): Inviting Comments and Suggestions on the Potential Development of a Challenge Prize for Transformative Genome Editor Delivery Technologies
Office of Strategic Coordination (Common Fund)
As part of the NIHs Somatic Cell Genome Editing (SCGE) program, the Office of Strategic Coordination (Common Fund) intends to publish a prize competition announcement to solicit entries for the multi-phase TARGETED (Targeted Genome Editor Delivery) Challenge.
The TARGETED Challenge aims to improve the current state of in vivo delivery technologies for genome editors in two Target Areas: 1) programmable delivery systems to deliver genome editing machinery that can target specific tissues or cell types, and 2) highly efficient non-viral delivery systems capable of crossing the blood brain barrier (BBB) to deliver genome editing machinery to a substantial proportion of clinically relevant cell types in the central nervous system (CNS).
This Notice is being provided to innovators of the upcoming opportunity to compete in the TARGETED Challenge. Publication of the Challenge announcement and launch of the submission portal are planned for May 2023. An informational webinar is planned for June 1st, 2023 at 12pm 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.
NIH intends to award a total prize purse of $6,000,000 which is anticipated to be awarded across the three phases of this competition. To be eligible to win prizes, participants must be led by a Team Leader who is a citizen or permanent resident of the United States or be an entity incorporated in and maintaining a primary place of business in the United States. The SCGE program is led by the NIH Common Fund, the National Center for Advancing Translational Sciences (NCATS), and the 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. Additional information about the planned prize competition is included below.
Recent advancements in the genome editing technology field have enabled scientists to manipulate genomic sequences rapidly and efficiently. Despite revolutionary progress in this area, several challenges remain. Existing gene editing technologies like CRISPR-cas9, base editors and prime editors have great potential, but existing delivery technologies are not able to deliver gene editing technologies to many target tissues and cell types in sufficient quantities, which hinders clinical applications. While some cell types, like hepatocytes in the liver, have many delivery technologies capable of delivering genome editors, there are many other organs and cell types that are harder to reach.
There is a strategic need for programmable delivery systems that can be modified to target distinct cells, tissues or organs, where the relationship between the modification to the delivery system and the altered specificity is well-understood, and where the editing performance for several delivery system configurations are at least as efficient as the state of the art for those targets.
There is also a strategic need for non-viral delivery systems capable of crossing the BBB. The BBB prevents unwanted substances from entering the extracellular fluid of CNS. It is composed of endothelial cells in the brain vasculature, as well as specialized glial cells (astrocytes) which surround blood vessels in the brain. The BBB blocks the uptake of many pharmaceuticals, including proteins and nucleic acids, hindering the development of treatments for brain-related diseases. An effective technology to deliver genome editing machinery across the BBB and into a substantial proportion of clinically relevant cell types would have broad implications for the treatment of many neurogenetic diseases.
Prize Competition Details
The TARGETED Challenge intends to offer a total prize purse of $6,000,000 which is anticipated to be distributed across three phases of the competition. Participants will be asked to submit proposals in Phase 1 and preliminary in vivo testing data from their solutions in Phase 2. Successful Phase 2 participants can enter their solution in Phase 3 for independent testing and validation in large animal models. NIH intends to offer cash prizes in each Phase to solutions in both Target Areas that meet the judging criteria.
The Office of Strategic Coordination intends to conduct this Challenge under the authority provided by Section 24 of the Stevenson-Wydler Technology Innovation Act of 1980 (15 U.S.C. 3719), as added by the America COMPETES Reauthorization Act of 2010 (Pub. L. 111-358).
SUBMISSIONS ARE NOT BEING SOLICITED AT THIS TIME.
Somatic Cell Genome Editing Program