Drug intelligence / Profile preview

deoxycytidine monophosphate + deoxythymidine monophosphate

Development stage
Preclinical
Lead developer
UCB
Modality
Small Molecules
Administration
Oral
01

Overview

This combination consists of two naturally occurring deoxyribonucleotides: **deoxycytidine monophosphate (dCMP)** and **deoxythymidine monophosphate (dTMP)**, both of which are fundamental building blocks of DNA. This combination has been studied as a pharmacological intervention for **thymidine kinase 2 (TK2) deficiency**, a rare mitochondrial DNA depletion syndrome. In TK2 deficiency, administration of these nucleotides is intended to bypass the defective TK2 enzyme, facilitating mitochondrial DNA synthesis and maintenance. This combination is considered investigational and has received orphan drug designation from the FDA for this indication, but has not been FDA approved[1][4][9].

Other names
dCMP + dTMPdeoxycytidylic acid + deoxythymidylic aciddeoxycytidylate + deoxythymidylate2'-deoxycytidine 5'-monophosphate + 2'-deoxythymidine 5'-monophosphatedeoxycytidine monophosphate + thymidine monophosphate
02

Targets

TK1 (Thymidine kinase 1)DCK (Deoxycytidine kinase)TK2 (Thymidine kinase 2)

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