Therapeutic focus​

Heritable disease targets

Many congenital disorders lack treatments and permanent cures. Using new and safer gene-therapy methods, mostly with viral vectors, some replacement therapy has been achieved. Replacing a missing gene has thus been made possible in targetable organs. Placing the new gene in a predestined location is another matter altogether. By cutting a gene in a desired location with the TGEE system it is now possible to do so. 

Furthermore, it will now be possible to replace, mutate, delete or insert a sequence into a malfunctioning or mutated gene that is associated with a genetic condition using the TGEE platform. There are  a multitude of different genetic diseases, many of these rare “orphan” diseases. TargetGene is primarily targeting monogenic dominant-negative disorders treatable ex-vivo by autologous re-transplantation of cells or tissues treated using the TGEE platform.

Cancer

Cancer has traditionally been considered by physicians as a multitude of different diseases. Recently, with the development of checkpoint inhibitors and CAR-T therapies, it is now likely that there may be common ways to treat multiple cancers by targeting not the cancer itself, but by improving the host immune response. Thus, while these marvelous new treatments are creating much hope, further improvements using gene-edited T-Cells are in the works, potentially enabling longer term remission or cure. Knockout of immune checkpoint receptor proteins, especially by using a more precise system such as the TGEE platform, would greatly enhance the long-term safety for the extended lifetime of the patient.

Immune disorders

Non-hereditary diseases such as autoimmune diseases are particularly applicable for curing via knockout or downregulation of the auto-antigen or by downregulation of an effector or an ancillary factor in an immune cell which affects or exacerbates the impact of the autoimmune disorder. TargetGene is utilizing the TGEE platform to target an orphan disease in this class.