NIH Names Winners for $6 Million Gene Editing Delivery Challenge

Rare Daily Staff

The National Institutes of Health named the winners of the second phase of its Targeted Genome Editor Delivery (TARGETED) Challenge, a competition designed to improve technologies for delivering genome editing tools to cells in the body.

The challenge is intended to support NIH’s Somatic Cell Genome Editing (SCGE) commitment to developing targeted delivery systems to deliver genome editors to somatic cells of the body. Somatic cell genome editing holds great promise in treating various diseases. However, current genome editing techniques, such as those based on CRISPR-Cas9, pose many obstacles that need to be overcome before they can be widely used in the clinic.

The TARGETED Challenge is designed to advance the current state of in vivo delivery technologies for genome editors in two target areas: Programmable Delivery System for Gene Editing, and Crossing the Blood-Brain Barrier. SCGE funds the challenge.

The phase 2 winners were selected based on experimental data demonstrating successful delivery and editing performance, along with innovative methodologies and technologies that effectively address the challenge of targeted gene editing delivery.

In addition to the monetary prize, each winner will advance to Phase 3 of the challenge, where they will have the opportunity to prepare their technologies for testing in large animal models through NIH-supported independent evaluation.

The winners for Phase 2 of the TARGETED Challenge for Programmable Delivery System for Gene Editing who were awarded $250,000 prizes include:

• Exosome Engineers (University of Nebraska-Lincoln) from Lincoln, NE and Durham, NC:

Editing the Genome in Any Tissue of Choice Through Programmable Milk Exosomes

• Perelman School of Medicine at the University of Pennsylvania from Philadelphia, PA:

Targeted Delivery of Genome Editing Machinery to Lungs, Systemic Endothelium, and Muscles David R. Liu Group, Broad Institute of MIT and Harvard
Tissue Specific Targeted eVLPs Through Barcoded Lentiviral Screening and Rational Engineering

• Beth Israel Deaconess Medical Center and University of Washington
  ENTER: Elastin-based Nanoparticles for Therapeutic delivERy, Self-Assembled Protein Nanoparticles for Targeted Gene Editor Delivery
• Ben Deverman Vector Engineering Laboratory, Broad Institute of MIT & Harvard
  Engineering Receptor-Targeted AAVs with Predictable Cellular and Species Tropism

In addition, $50,000 prizes were awarded to:

• Columbia-Leong Oral Editing
  Oral Nonviral Gene Editing System
• Helex, Inc.
  EPIC-Cure platform

●  Medical College of Wisconsin and Università degli Studi di Milano
    One System, Any Target

• ProEdit (University of Washington)
  ProEdit: Programmable Genome Editing to Target Diverse Organ and Cell Lineages
• Smart Gene Darts (Case Western Reserve University)
  Smart Programmable Lipid Nanoparticle Platform for Gene Editing

In Target Area 2: Crossing the Blood-Brain Barrier, $250,000 prizes were awarded to:

● Crisaptics Trans-BBB Genome Editing Team (University of Maryland School of Medicine)
   Crisaptics Trans-BBB

• PERCEPT (Innovative Genomics Institute at UC Berkeley)
  Chemically engineered CRISPR enzymes for accessible whole-brain genome editing
• Icahn School of Medicine at Mount Sinai
  Blood-brain barrier-crossing lipid nanoparticles for genome editing
• STEP Team (Yale University)
  BRAIN TARGETED-STEP RNPs for Delivery of Genome Editing to the Brain

The Phase 3 of the challenge will be divided into two parts. In Phase 3a, winners will demonstrate that their technology is ready for large animal testing. Those who successfully complete Phase 3a will receive additional prize funding and advance to Phase 3b, where their technologies will undergo independent testing and validation in large animal models. The top-performing solutions in Phase 3b will be awarded up to $625,000.

The TARGETED Challenge is expected to conclude in 2027, with the announcement of the final winners who successfully demonstrate their ability to deliver genome editing tools effectively to target tissues.