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ADX-324
ADX-324 is an ADAR enzyme activator that enhances adenosine-to-inosine RNA editing to modulate gene expression and protein function.
ADX-324 is an ADAR enzyme activator that enhances adenosine-to-inosine RNA editing to modulate gene expression and protein function. Used for Duchenne muscular dystrophy (DMD).
At a glance
| Generic name | ADX-324 |
|---|---|
| Also known as | siRNA |
| Sponsor | ADARx Pharmaceuticals, Inc. |
| Drug class | ADAR activator |
| Target | ADAR1/ADAR2 |
| Modality | Small molecule |
| Therapeutic area | Genetic/Rare Disease |
| Phase | Phase 3 |
Mechanism of action
ADX-324 works by activating ADAR (adenosine deaminase acting on RNA) enzymes, which catalyze the conversion of adenosine to inosine in RNA molecules. This RNA editing process can alter the amino acid sequence of proteins or affect RNA stability and localization, potentially correcting disease-causing mutations or modulating pathogenic protein function. This represents a novel approach to treating genetic and acquired diseases through post-transcriptional RNA modification.
Approved indications
- Duchenne muscular dystrophy (DMD)
Common side effects
Key clinical trials
- Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients (PHASE1, PHASE2)
- Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE) (PHASE3)
- STOP-HAE: A Phase 3 Study of ADX-324 in HAE (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ADX-324 CI brief — competitive landscape report
- ADX-324 updates RSS · CI watch RSS
- ADARx Pharmaceuticals, Inc. portfolio CI