Payload Information

General Information of This Payload
Payload ID
PAY0JRATG
Name
DUX4 siRNA
Synonyms
DUX4 siRNA
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Target(s) Double homeobox protein 4 (DUX4)
 Target Info 
The activity data of This Payload
Standard Type Value Units Cell line Disease Model Cell line ID Reference
Methylation Fold Change ≈2.5 .
FMUFAHi001-A cells
Facioscapulohumeral dystrophy
CVCL_C0FU 
[1]
Each Antibody-drug Conjugate Related to This Payload
Full Information of The Activity Data of The ADC(s) Related to This Payload
AOC-1020 [Phase 2]
 ADC Info 
Identified from the Human Clinical Data
Click To Hide/Show 1 Activity Data Related to This Level
Experiment 1 Reporting the Activity Date of This ADC [2]
Related Clinical Trial
NCT Number NCT05747924  Phase Status Phase 1
Clinical Description
A randomized, double-blind, placebo-controlled, phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of AOC 1020 administered intravenously to adult participants with facioscapulohumeral muscular dystrophy (FSHD).
References
Ref 1 DICER/AGO-dependent epigenetic silencing of D4Z4 repeats enhanced by exogenous siRNA suggests mechanisms and therapies for FSHD. Hum Mol Genet. 2015 Sep 1;24(17):4817-28.
Ref 2 A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD), NCT05747924