Etranacogene dezaparvovec

PreclinicalGene therapy

Etranacogene dezaparvovec is a gene therapy that works as a Factor IX Gene Therapy.[1] It is being investigated across 1 indication, with its most advanced program in Preclinical.

Etranacogene dezaparvovec is one of 2 gene therapy programs targeting Hemophilia B. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[2]

Indication

Hemophilia BUnknown

Hemophilia BPreclinical

Unknown

Competition

Competitive Landscape

6 competitors in Hemophilia B

#DrugCompanyPhaseTrials

BT200

BBand Therapeutics

Phase 3

1 2

Etranacogene dezaparvovec

CSL Behring

Preclinical

—

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Sources

References

[1]
ChEMBL — Etranacogene dezaparvovec compound record. European Bioinformatics Institute (EMBL-EBI).
[2]
Open Targets Platform — Drug–disease associations for Etranacogene dezaparvovec. EMBL-EBI & Wellcome Sanger Institute.
[3]
PubMed — 10 publications indexed for Etranacogene dezaparvovec. U.S. National Library of Medicine.
[4]
SEC EDGAR — CSL Behring company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Factor IX Gene Therapy[1]
Modality: Gene therapy[1]
Highest Phase: Preclinical
Company: CSL Behring

Key Publications[3]

Trismus and Health-Related Quality of Life in HNC Patients up to 10 Years Post-Radiotherapy Completion.

Head NeckSep 3, 2026DOI: 10.1002/hed.70442

[Design and implementation of clinical trials for new TCM based on human use experience].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260512.501

[Therapeutic mechanism of Astragalus polysaccharides in bladder dysfunction in DBA mice based on regulatory role of myosin Va in neurotransmission].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260429.401

[Mechanism study on baicalein inhibiting cervical cancer driven by persistent HPV infection via restoring MAVS-IRF3-IFN-β mitochondrial antiviral signal].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260330.704

[Study on mechanism of Wendan Decoction in intervening in nonalcoholic fatty liver disease based on proteomics and network pharmacology].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260414.703

[Tongmai Yangxin Pills inhibit inflammation and apoptosis through AGE-RAGE signaling pathway and improve cardiac arrhythmia in ischemia-reperfusion rats].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260414.702

[Efficacy and safety of Salvianolic Acid for Injection treatment during recovery phase of ischemic cerebrovascular disease: clinical study based on single-arm objective performance criteria method].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260514.501

[Astragali Radix enhances radiotherapy efficacy in lung cancer by modulating SPOP-mediated homologous recombination repair].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260506.406

[Effect and mechanism of Modified Qinghao Biejia Decoction medicated serum in inducing ferroptosis in acute myeloid leukemia cells based on TFR1/ACSL4/LPCAT3 signaling axis].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260506.401

[Research progress in nano-delivery systems of active ingredients in TCM for treating kidney diseases].

Zhongguo Zhong Yao Za ZhiAug 1, 2026DOI: 10.19540/j.cnki.cjcmm.20260430.303

Etranacogene dezaparvovec

Status by Indication[2]

Competitive Landscape

References

Drug Info

Quick Facts

Key Publications[3]

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