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
View the Phase 2 Winners
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