National Institutes of Health Announces Phase 1 Winners of the $6M TARGETED Challenge

Dec 13, 2023

We are thrilled to announce the Phase 1 winners of the TARGETED (Targeted Genome Editor Delivery) Challenge! The competition garnered 54 proposals from diverse independent research groups, universities, companies, and laboratories, spanning both target areas: Programmable Delivery System for Gene Editing and Crossing the Blood-Brain Barrier.

This three-phase challenge represents a US$6M initiative supporting the NIH's Somatic Cell Genome Editing (SCGE) commitment to developing targeted delivery systems for delivering genome editors to somatic (non-reproductive) cells in the body.

In Phase 1, participants submitted proposals outlining their solutions and how they intend to meet the requirements for one of the Target Areas.

Phase 1 winners will be invited to Phase 2 to further advance their research and provide data from experimental testing demonstrating delivery and editing performance. They will also describe their methodology, technology, and how their solution addresses the Challenge criteria.

Phase 2 is open to new participants, and entry is possible without having participated in the previous phase. Up to 10 winners of Phase 2 will each be awarded US$250,000 and will be eligible to compete in Phase 3.

Target Area 1: Programmable Delivery System for Gene Editing

Winning Solutions $75,000 USD Prize

Perelman School of Medicine at the University of Pennsylvania: Targeted Delivery of Genome Editing Machinery to Blood Marrow Stem Cells, Pulmonary Endothelium, and Cardiomyocytes

Nizar Y. Saad, PhD and Meisam Naeimi Kararoudi, PhD: Development of A Programmable Extracellular Vesicle-Based Delivery System of Allele-Specific Gene Editing Therapy Using Bioorthogonal Chemistry

Beth Israel Deaconess Medical Center: Self-Assembling Protein Polymer Micelles for Targeted Gene Editor Delivery

Reza Shahbazi, Ph.D. (Lead PI) Indiana University School of Medicine: Developing In Vivo CRISPR Nanotherapeutics with Enhanced Safety and Precision by Dual Targeting Approach

Vesigen Therapeutics: Engineered ARMMs: Promising Human-Derived Vectors for Cell Type-Specific Delivery of Genome Editors

Meritorious Solutions $50,000 USD Prize

GenEdit, Inc.: Programmable Delivery of Genetic Medicine to Multiple Tissues with Polymer-based Hydrophilic Nanoparticles

Stanley Qi lab: TRANSFER: Data-driven programmable macromolecule delivery via trans-endocytosis

Columbia University: Development of an Orally-Administered, Nonviral Gene Editing System for the Gastrointestinal Tract, Mesenteric Lymph Node, and Liver

GENOMIXIO: Programmable Editing of APOE Gene Using PEG-LNPs

Meritorious Solutions $25,000 USD Prize

Carmine Therapeutics, Inc.: Red Blood Cell Extracellular Vesicle as the ideal delivery system for genome editing

Broad Institute of MIT & Harvard: A platform for targeting AAVs to specific receptors

Dehua Pei, PhD Ohio State University: Intracellular Delivery of Gene-Editing Enzymes with Membrane Translocation Domains

David R. Liu Group Broad Institute of MIT and Harvard: Targeted eVLPs through barcoded lentivirus screening

SiVEC Biotechnologies, Inc.: A Vehicle-Encoded Molecular Targeting System for Delivery to Precise Cells and Tissues

Helex, Inc.: Proprietary double lock system for targeted LNP delivery and tissue specific gene editing apparatus for safer in vivo gene editing therapeutics

ExoTarget Team (University of Florida): ExoTarget Platform as a programmable delivery system

Hunterian Medicine LLC: Programmable Gene Editing with Tissue- and Cell-specific Promoters

Battelle Memorial Institute: TA1 Phase 1: High throughput in vivo screening of PNPs for Programmable delivery of gene editors

Orion Therapeutics, Inc.: Multifunctional Lipid Nanoparticle Delivery System for Targeted Delivery of CRISPR Therapeutics

Exosome Engineers (University of Nebraska): Editing the genome in any tissue of choice through programmable milk exosomes

Target Area 2: Crossing the Blood Brain Barrier

Winning Solutions $75,000 USD Prize

ARENEX (University of Kentucky Research Foundation): Artificially engineered exosomes for gene editing in the brain

ZhouTeam@Yale: TARGETED-STEP RNPs for delivery of genome editing to the brain

Cornell University: Systemic and targeted delivery using engineered endogenous virus-like vesicles for gene editing in the central nervous system

Columbia University: Focused Ultrasound-Assisted Intravenous Delivery of Optimized Lipid Nanoparticles for Targeted Brain Gene Editing

LEDGE Team: Delivery of gene editing complex across the BBB using lectin-mediated transport

Meritorious Solutions $50,000 USD Prize

Orion Therapeutics, Inc.: Multifunctional Lipid Nanoparticle Delivery System for Delivery of CRISPR Therapeutics Across the Blood-Brain Barrier

Meritorious Solutions $25,000 USD Prize

Drs. Wenhui Hu and Qingsheng Li: Receptor-mediated and exosome-decorated lentivirus-like particles traverse blood-brain barrier for brain gene editor delivery

Jiukuan Hao & Bin Guo, College of Pharmacy at University of Houston: Targeting Blood-Brain Barrier for Crispr/Cas9 Brain Delivery

Talinted, Inc.: Exosome Mediated Targeting of Capsid Bound Gene Editing Material

Battelle Memorial Institute: Polymeric nanoparticle-facilitated delivery of gene editors across the blood brain barrier