Discovery of a METTL3 inhibitor

Tech ID:

METTL3 Inhibitors as Leukemia therapeutics

THE PROBLEM Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a relatively rare cancer (1% of all cancer incidence cases), however it accounts for a disproportionally high number of cancer-related deaths. AML’s lethality can be contributed to its fast progression, low survivability, and high rate of relapse.

Furthermore, AML is a heterogeneous disease with distinct morphologies, epigenotypes/genotypes. AML has at least two major subtypes – acute promyelocytic leukemia and secondary AML. However, new research has showed it is actually an umbrella of at least 11 different diseases with many different causes and genetic themes. This diverse pathophysiology makes AML difficult to research and treat.

Value Proposition

Unmet Needs

•       Need drugs that are less toxic for use in elderly patients

•       Need new therapies which improve overall survival, rather than marginally improving remission, due to AML’s high relapse rates

•       Drugs that can be used to treat a variety of the numerous varieties and subtypes of AML

Advantages of the new therapeutic molecules

•       Relatively novel target, METTL3

•       These small molecules has not be previously indicated for treatment of AML through inhibition of METTL3

•       Mechanism of inhibition that may be therapeutic across many different AML subtypes.



Stage of Development

The inventor, through the combined use of both a crystal structure of the METTL3-MTase core and in-silico analysis has identified a series of inhibitory compounds. One candidate compound was able to inhibit the methyltransferase activity of the METTL3 subunit by four fold in the inventor’s in vitro assay. The inventor is now probing the specificity, potency and tumor cell killing ability of these compounds and derivative formulations in vivo.




Small Molecule
For information contact:
File Closed
Yogesh Gupta
Patent Information: