University of Pittsburgh

Novel Targeted Gene Therapy for Cancers

Scientists from University of Pittsburgh have developed a novel approach to target cancer cells through the insertion of a “suicide gene” (HSV1-tk) into key gene fusion areas of the cancer genome.

Description

Targeting gene fusion points remains an elusive goal in cancer therapy. This novel approach can insert HSV1-tk at specific fusion points and could lead to improved survival in cancer patients through initiation of cell death or metastasis inhibition, personalized to each patient’s unique cancer.

Advantages

• Research has identified a panel of fusion genes present in various cancers including hepatocellular carcinoma (HCC)
• Using a tailored Cas9-based genome editing approach it is possible to selectively insert the gene HSV1-tk to the fusion point
• Creation of this unique breakpoint, present only in cancer tissue provides a target for therapeutic intervention which is less likely to result in drug resistance through gene mutation
• This approach has been studied in TMEM135-CCDC67 in human prostate cancer highlighting this potential of genome editing to provide a genotype-specific approach to treat various cancers.

Invention Readiness

Two fusion gene breakpoints have been successfully targeted with an HSV gene. Treatment with antivirals led to cell death in vivo and improved mortality in vitro. Further work is required to develop clinical feasibility and identify other potential targets or insertion genes.

IP Status

https://patents.google.com/patent/US10822622B2