An introduction to a novel drug class


Similar to other fundamental therapeutic approaches like gene editing and gene therapy, the ability to drug harmful epitranscriptomics modifications could lead to a plethora of novel treatment options and potentially cures for a variety of diseases.


Epitranscriptomics in Cancer

Epitranscriptomics modifications play a critical factor in cell differentiation and stem cell pluripotency. The fact that cancer is based on the misregulation of cell growth and differentiation points towards the possibility that cancer cells may have hijacked the epitranscriptomics machinery to enhance their survival. Several studies have demonstrated roles for demethylation or lack of methylation in promoting cancer progression. Developing drugs reversing tumor-promoting epitranscriptomic modifications or activating tumor-suppressing epitranscriptomic regulatory pathways may represent a promising therapeutic strategy to treat cancers.

Beyond cancer, epitranscriptomics modifications are implicated to play a role in auto-immune and neurodegenerative disorders, viral infections and other indications. Gotham will focus initially on targets with the most compelling links to disease while research activities aimed at unlocking the full potential of this emerging sector will continue at Gotham and its research partners.