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Targeted Genome Editor Delivery (TARGETED) Phase 1 Challenge Winners

The National Institutes of Health, through its Somatic Cell Genome Editing program, launched this groundbreaking $6 million challenge to improve gene editing technology by specifically developing targeted delivery systems of genome editors to somatic (non-reproductive) cells in the body.

See more details about the Challenge

Winners of Phase 1

Target Area 1: Programmable Delivery System for Gene Editing

Winning Solutions $75,000 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
  • ExoThera Bio      
    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 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 Prize

  • Carmine Therapeutics, Inc.      
    Red Blood Cell Extracellular Vesicle as the Ideal Delivery System for Genome Editing
  • Broad Institute of MIT and Harvard      
    A Platform for Targeting AAVs to Specific Receptors
  • Dehua Pei, Ph.D., 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 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 Prize

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

Meritorious Solutions $25,000 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 and 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


See registration details for the launch of Phase 2 of the $6 million TARGETED Challenge

Last updated on December 22, 2023