Payload Information

General Information of This Payload
Payload ID
PAY0JRATG
Name
DUX4 siRNA
Synonyms
DUX4 siRNA
   Click to Show/Hide
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 1/2]
 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

If you find any error in data or bug in web service, please kindly report it to Dr. Shen et al.