Drugs / RAG-21
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 0 | NCT07080801 | Aug 2025 → Aug 2026 overdue | amyotrophic lateral sclerosis | Beijing Tiantan Hospital | Not yet recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2024-11-19 | Ractigen Therapeutics. | Regulatory Ractigen Therapeutics Announces FDA Orphan Drug Designation for RAG-21 for the Treatment of ALS ractigen.com ↗
Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy targeting the FUS gene, for the treatment of amyotrophic lateral sclerosis (ALS). |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2024-11-19 | Ractigen Therapeutics. | Ractigen Therapeutics Announces FDA Orphan Drug Designation for RAG-21 for the Treatment of ALS ractigen.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RAG-21 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07080801 ↗ |
| Action | Inhibit | “mediates its degradation, thereby downregulating FUS gene expression” NCT07080801 ↗ |
| Modality | Small interfering RNA (siRNA) | “RAG-21 is a small interfering ribonucleic acid (siRNA)” NCT07080801 ↗ |
| Route | Intrathecal | “intrathecal bolus administration of RAG-21” NCT07080801 ↗ |
| Target | FUS | “it targets the FUS gene” NCT07080801 ↗ |