Drug intelligence / Profile preview

YTHDC1 inhibitor (Transition Bio)

Development stage
Preclinical
Lead developer
Transition Bio
Modality
Small Molecules
Administration
Oral
01

Overview

Transition Bio's YTHDC1 inhibitor program focuses on developing small molecule inhibitors that target the YTH domain-containing protein 1 (YTHDC1). YTHDC1 is an m6A "reader" protein that, in certain cancers like acute myeloid leukemia (AML), localizes with m6A-marked RNAs in phase-separated biomolecular condensates. These condensates stabilize oncogenic transcripts, most notably MYC and BCL2, promoting tumor cell proliferation and survival. By blocking the interaction between YTHDC1 and m6A-modified RNAs, these small molecules disrupt the formation of these condensates, leading to the downregulation of MYC and other oncogenic drivers. The program is currently in the IND-enabling/lead optimization phase, with potential applications in MYC-driven cancers, including AML, prostate cancer, and small cell lung cancer.

Other names
YTHDC1-MYC programYTHDC-1-MYC programYTHDC 1-MYC programYTHDC1 inhibitorsYTHDC-1 inhibitorsYTHDC 1 inhibitors
02

Targets

YTHDC1

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