Target intelligence / Profile preview

DEAD-box helicase 3 X-linked (DDX3X) (DDX3X)

Target
DDX3X
Molecular classification
Enzyme, RNA helicase, DEAD-box helicase family, Superfamily 2 (SF2) helicase
01

Overview

DEAD-box helicase 3 X-linked (DDX3X) is a multifunctional ATP-dependent RNA helicase belonging to the DEAD-box protein family [4, 9]. It plays a central role in nearly all aspects of RNA metabolism, including transcription, pre-mRNA splicing, mRNA export, and translation initiation, particularly for mRNAs with complex 5' untranslated regions [11, 12]. Beyond its core RNA-processing functions, DDX3X is a key regulator of cellular signaling pathways such as Wnt/beta-catenin and NF-kappaB, and it is involved in the formation of stress granules and the innate immune response [5, 10, 18]. In oncology, DDX3X is frequently overexpressed and acts as an oncogene in various cancers, including breast, colorectal, and glioblastoma, where it promotes cell proliferation and metastasis [1, 13, 15]. Conversely, it can act as a tumor suppressor in certain contexts, such as in some cases of hepatocellular carcinoma [25, 27]. DDX3X is also a critical host factor for the replication of numerous viruses, including HIV-1, HCV, and SARS-CoV-2, making it a promising target for broad-spectrum antiviral therapy [6, 10, 29]. Small molecule inhibitors like RK-33 and BA103 are being developed to target its ATPase or helicase activities to treat cancer and viral infections [1, 31].

Other names
DDX3DBXHLP2DDX14CAP-RfMRX102MRXSSBDEAD-box polypeptide 3 X-linkedATP-dependent RNA helicase DDX3X
02

Mechanism of action

Inhibition of the ATP-binding site (ATPase activity) or the RNA-binding site (helicase activity), leading to suppressed mRNA translation of oncogenic proteins, disruption of Wnt/beta-catenin signaling, and induction of the innate immune response [1, 13, 15, 30].

03

Biological functions

mRNA translation initiation (5' UTR scanning) [4, 11, 12]Alternative pre-mRNA splicing [4, 9, 26]mRNA nuclear export [4, 12]Transcription regulation [4, 9]Wnt/beta-catenin signaling pathway activation [1, 7, 22]NF-kappaB signaling regulation [10, 17]Stress granule formation and dynamics [4, 5, 10]Innate immune response (RIG-I and NLRP3 pathways) [5, 6, 18]
04

Disease associations

Cancer (Breast, Colorectal, Medulloblastoma, Lymphoma, Glioblastoma, Oral, Lung, Prostate) [1, 2, 7, 13, 15, 22, 24, 31]Viral infection (HIV-1, HCV, HBV, SARS-CoV-2, Zika, West Nile virus) [6, 10, 12, 29]Neurodevelopmental disorder (DDX3X syndrome/Intellectual disability) [8, 19, 32]
05

Safety considerations

Potential for systemic toxicity due to the essential role of DDX3X in normal cell survival and RNA metabolism [2, 13]Risk of neurotoxicity or developmental defects given its critical role in brain development [8, 19]Context-dependent tumor-suppressive functions that could lead to adverse effects in certain tissues if inhibited [25, 27]Off-target effects due to the high conservation of the ATP-binding site across the DEAD-box helicase family [13, 30]
06

Interacting drugs

RK-33 [1, 7, 29]

6 more in the full profile.

07

Biomarkers

DDX3X protein expression levels (IHC) [1, 7, 24]Nuclear beta-catenin localization [1, 7, 14]DDX3X somatic mutations (e.g., in medulloblastoma or Burkitt lymphoma) [2, 22, 33]Stress granule presence [4, 22]

Beyond the preview

Go deeper on DEAD-box helicase 3 X-linked (DDX3X) (DDX3X).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on DEAD-box helicase 3 X-linked (DDX3X) (DDX3X).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call