Ultra-precise gene editing platform seeking gene and cell therapy applications.

TargetGene is a genome-engineering company offering its ultraprecise, radically safer TGEE gene-editing platform for cell genetic engineering. TGEE is an obligatory dimeric system utilizing two guide RNAs, instead of CRISPR's one, and is thus expected to have no off-target mutations. TGEE can thus potentially be used in vivo, off-the-shelf or ex-vivo to enable genetic manipulation for treatment of heritable or prevention of acquired diseases.

TargetGene, founded in 2012 in Israel, is a pioneer in the gene-editing field, propelled by its proprietary best-in-class platform known as T-GEE. T-GEE enables SAFE modification of the patient’s own DNA. With T-GEE, genes can be added, deleted, or precisely replaced—effectively rewiring the transcriptome. We thus intend to correct genetic abnormalities or enhance specific cell types, ultimately leading to cures rather than chronic treatments.

TargetGene has an exceptionally rich and broad patent portfolio including >15 granted patents and multiple patents pending. We are strategically partnering in different cell therapies and are also key partners in CAR-T REX, a major EU-funded consortium focused on CAR-T therapies for solid tumors.

Our novel and proprietary T-GEE programmable molecular “DNA scissors” utilizes two guides and an obligatory dimeric nuclease to achieve its exquisite specificity. T-GEE solves the biggest problem of gene editing - off-target mutation of unintended genes- and is predicted to be orders of magnitude more precise than CRISPR-Cas9, potentially creating a radically safer therapeutic tool.

The flexibility of T-GEE genome editing and the insertion of any payload DNA to almost any specific site can pave the way for personal correction of genetic disorders. Use of T-GEE allows precise insertion of promoter-free genes, CARs and regulatory elements into cells without use of viruses, potentially reducing costs and production time and significantly improving safety for the patient thus benefiting public health and society.

TargetGene is "rewiring" cells by regulating multiple gene expression with a single gene-editing event with a novel, patented method. The cell-product thus contains an auto-regulated genetic circuit controlled by microRNAs (miRNAs).  TargetGene's rewired T-cells are currently being used to block T-cell exhaustion in the tumor for the CAR T-REX project and are being adopted to other cells and needs.