Ljungman receives $500K from the Little Warrior Foundation for Ewing sarcoma gene-editing therapy

Mats Ljungman, Ph.D., professor of radiation oncology and environmental health sciences at the University of Michigan, received a $528,728 award from the Little Warrior Foundation to further research using a gene editing system to target a gene fusion involved in Ewing sarcoma.

Ljungman and his laboratory have developed KLIPP (the Swedish word for “cut”), a next-generation CRISPR-based therapeutic platform designed to target cancer-specific structural variant junctions (gene fusions).

The fusion gene driving Ewing sarcoma, most commonly EWS::FLI1, is an ideal target for this strategy because it is a non-hereditary mutation found only in tumor cells, allowing for highly selective targeting.

KLIPP utilizes guide RNAs that bind to precise DNA sequences on both sides of the fusion breakpoint. Only when both guide RNAs bind in close proximity is the Fok1 enzyme activated, enabling highly selective cutting of the cancer-driving fusion DNA. Without this genetic blueprint intact, the corresponding oncogenic protein can no longer be produced.

The funding will support the development of a weaponized lipid nanoparticle delivery system to more efficiently deliver KLIPP to tumor cells, followed by validation of this therapeutic strategy in both in vitro and in vivo Ewing sarcoma models.