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