Delivering the Future Targeted Genomic Medicine
Sign up here to be part of this groundbreaking challenge that could shape the future of genome editing and gene therapy.
Overview
The National Institutes of Health (NIH) has launched the TARGETED (Targeted Genome Editor Delivery) Challenge. This competition is part of 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. This multi-phase Challenge launched in May with $6M USD in prize money.
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 nonviral 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).
NIH will award up to $6M USD in prize money and provide independent testing for the most promising delivery vehicles in two Target Areas:
1) Programmable delivery systems and
2) Nonviral delivery systems crossing the blood brain barrier.
Top competitors could win $1M in prize money and have their solution
independently tested and validated.
Gene editing holds the promise to treat genetic diseases at the source by correcting the faulty genetic patterns within our cells. 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. There is also a strategic need for nonviral delivery systems capable of crossing the BBB, a structural and functional barrier that prevents unwanted substances from entering the extracellular fluid of CNS. The BBB also blocks the uptake of many pharmaceuticals, including proteins and nucleic acids, hindering the development of treatments for brain-related diseases.
A broad range of delivery vehicles are of interest, including but not limited to lipid nanoparticles, liposomes, exosomes, antibody-like technologies, peptide carriers, microbe-based technologies, inorganic delivery systems, polymer-based systems, nucleic acid-based systems, and micro and nanobots.
Target Areas
NIH has chosen two Target Areas with the potential to result in transformative, strategic advances. While all solutions must solve one of the Target Areas, solutions can also address both 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 the current state of the art. 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
Solutions to Target Area 2 should be a highly efficient nonviral delivery system capable of crossing the BBB to deliver genome editing machinery to a majority of target cell types in the central nervous system (CNS). Programmable solutions that only target CNS targets should be submitted under Target Area 2. Solutions that meet the requirements for Target Area 2 but are also programmable to target a non-brain organ may be submitted for consideration in both Target Areas but will only be eligible for one prize.
Who Can Compete?
The TARGETED Challenge is open to qualified groups of individuals which are led by a Team Leader who is a citizen or permanent resident of the United States, or teams from organizations or institutions that are incorporated in and maintaining a primary place of business in the US. NIH hopes to attract participants from within the genome editing field, and from delivery technology developers who have not previously delivered genome editors but have delivered other macromolecules.
How Will the Competition Unfold?
PHASE 1: PROPOSALS
- Start Date: May 2023
- Submission Deadline: October 5, 2023
Innovators must submit a proposal describing their solution and how it will address the requirements for one of the Target Areas. Innovators may propose solutions to both Target Areas but must submit separate proposals. Winners will be awarded cash prizes and are expected to participate in Phase 2. Technologies in more advanced development stages may bypass Phase 1 and enter directly at Phase 2.
PHASE 2: PRELIMINARY DATA
- Estimated Start Date: December 2023
- Estimated Submission Deadline: January 2025
Phase 2 is open to Phase 1 winners and all eligible solvers. Participants must address how the solution meets the solution requirements for that Target Area, provide detailed information about in-house experimental results, and provide a description of the methodologies used (including frameworks used to ensure appropriate animal protections for any animal experiments included in the submission). Winners will win cash prizes and advance to Phase 3 based on the Judging Criteria.
PHASE 3: FINAL DATA, INDEPENDENT TESTING, AND VALIDATION
- Estimated Start Date: March 2025
- Submission Deadline: TBD
Only innovators selected to advance from Phase 2 will be eligible to participate in Phase 3. For Phase 3a, innovators demonstrate that their technology is ready for NIH-sponsored large animal testing. Selected teams will then prepare for reagent scale up and protocol development for 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.
Sign up here to be part of this groundbreaking challenge that could shape the future of genome editing and gene therapy.
