Drugs / RAG-17
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 02 trials · 1 met primary | ||||||
| Phase 0 | NCT07077668 | Jul 2025 → Dec 2026 expected | amyotrophic lateral sclerosis | Beijing Tiantan Hospital | Not yet recruiting | No outcome recorded |
| Phase 0 | NCT05903690 | May 2023 → Jul 2024 | — | Beijing Tiantan Hospital | Completed | Met primary |
| Phase 11 trial | ||||||
| Phase 1 | NCT06556394 | Dec 2024 → Feb 2026 overdue | amyotrophic lateral sclerosis | Ractigen Therapeutics. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-07-15 | Ractigen Therapeutics. | Results Ractigen Therapeutics Announces Landmark Publication in Nature Medicine Highlighting Unprecedented Preclinical Efficacy and Positive First-in-Human Clinical Data for RAG-17 in SOD1-ALS ractigen.com ↗
First-in-Human trial met primary safety endpoints with no Serious Adverse Events (SAEs); demonstrated mean reductions of up to 69% in CSF SOD1 protein and 62% in plasma NfL |
| 2026-04-21 | Ractigen Therapeutics. | Results Ractigen Therapeutics Presents Positive Phase I Data for RAG-17 in SOD1-ALS at the 2026 AAN Annual Meeting, Demonstrating 81% Reduction in Neurofilament Light Chain ractigen.com ↗
Ractigen Therapeutics, a clinical-stage biopharmaceutical company focused on developing next-generation RNA-based medicines, today announced positive preliminary data from the single ascending dose (SAD) Phase I portion of its ongoing Phase I/II clinical trial of RAG-17. |
| 2026-04-19 | Ractigen Therapeutics. | Results Safety, Biomarker Responses, and Preliminary Efficacy Following a Single Intrathecal Dose of RAG-17, a Novel siRNA Therapeutic for SOD1-ALS: Results from an Ongoing Phase I/II Study (Poster) ractigen.com ↗
Ractigen Therapeutics Presents Positive Phase I Data for RAG-17 in SOD1-ALS at the 2026 AAN Annual Meeting, Demonstrating 81% Reduction in Neurofilament Light Chain |
| 2025-04-09 | Ractigen Therapeutics. | Results RAG-17, a Novel siRNA Therapy for SOD1-ALS: Safety and Preliminary Efficacy from a First-in-human Trial (Abstract) ractigen.com ↗ |
| 2025-04-09 | Ractigen Therapeutics. | Results Ractigen’s RAG-17 ALS Data Shines at AAN 2025: Earns Top Award, Phase I Advances ractigen.com ↗
Final results from an Investigator-Initiated Trial presented for the first time, demonstrating favorable safety, significant target engagement, and encouraging clinical & biomarker signals |
| 2024-09-10 | Ractigen Therapeutics. | Results Ractigen Announces Positive Clinical Data for RAG-17 in ALS-SOD1 Treatment from Investigator-Initiated Trial ractigen.com ↗
The study revealed encouraging results in the treatment of amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations (ALS-SOD1). |
| 2024-05-15 | Ractigen Therapeutics. | Regulatory Ractigen Therapeutics Receives IND Approval from China’s NMPA to Initiate Phase 1 Clinical Trials for RAG-17 in SOD1-ALS Patients ractigen.com ↗
This marks a pivotal moment for our company, as RAG-17 is our first siRNA drug targeting the central nervous system to receive CDE approval. |
| 2023-03-03 | Ractigen Therapeutics. | Regulatory Ractigen Therapeutics Receives FDA Orphan Drug Designation for the novel oligonucleotide conjugate RAG-17 for the Treatment of Amyotrophic Lateral Sclerosis (ALS) ractigen.com ↗
Jiangsu, China — Ractigen Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RAG-17, a novel siRNA modality for the treatment of Amyotrophic Lateral Sclerosis (ALS). |
All press releases naming this drug 15 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RAG-17 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05903690 ↗ |
| Action | Inhibit | “intrathecal RAG-17 led to dose-dependent, durable reductions in SOD1 mRNA (CNS) and protein (cerebrospinal fluid (CSF)).” PMID 42458007 ↗ Jul 2026 |
| Modality | Small interfering RNA (siRNA) | “RAG-17, an siRNA-targeting SOD1” PMID 42458007 ↗ Jul 2026 |
| Route | Intrathecal | “intrathecal RAG-17 led to dose-dependent, durable reductions in SOD1 mRNA (CNS) and protein (cerebrospinal fluid (CSF)).” PMID 42458007 ↗ Jul 2026 |
| Target | SOD1 | “RAG-17, an siRNA-targeting SOD1” PMID 42458007 ↗ Jul 2026 |