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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

Issued by

Office of Strategic Coordination (Common Fund)

Purpose

As part of the NIH’s 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 1^st, 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.

Background

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.

1. Phase 1: Innovators participating in this Challenge will submit a proposal describing their proposed solution and how it will address the requirements for one of the Target Areas. Innovators may submit proposed solutions to both Target Areas but must do so with separate proposals that independently address each Target Area’s requirements. Selected winners will win cash prizes and are expected to participate in Phase 2. Participation in Phase 1 is not a requirement for participation in Phase 2; however, it is strongly encouraged.
    
2. Phase 2: Phase 2 is open to Phase 1 winners and all other eligible solvers. In this phase, innovators must submit data from in vivo testing in small mammals and describe their methodology, technology, and how their solution addresses the Challenge.  Selected winners will win cash prizes and advance to Phase 3 based on the Judging Criteria. 
    
3. Phase 3: Only innovators selected to advance from Phase 2 will be eligible to participate in Phase 3, which is separated into Phase 3a and 3b. All participants must submit solutions for Phase 3a to be eligible to participate in Phase 3b. For Phase 3a, innovators must demonstrate that their technology is ready for large animal testing. Selected teams will then prepare for reagent scale up and protocol development for large animal testing. Innovators who submit their reagents and protocols by the Phase 3b deadline will be awarded NIH-funded independent, large animal testing to validate their solution. NIH will review the results of the independent validation and innovators whose solutions meet or exceed the Judging Criteria across all Phases could be eligible for a total award of up to $1million.

The Challenge announcement will likely be posted on multiple websites, including Challenge.gov and the SCGE program website, and broadly disseminated across NIH stakeholder networks.

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.

Inquiries

Please direct all inquiries to:

Somatic Cell Genome Editing Program
Email: SCGEprogram@od.nih.gov