Drugs / RAG-18
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RAG-18

Small activating RNA (saRNA)

Developed for
Duchenne muscular dystrophy
Investigated by
Peking Union Medical College Hospital · Ractigen Therapeutics.

Trials 1

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 0 NCT07282652 Dec 2025 → Nov 2026 expected Duchenne muscular dystrophy Peking Union Medical College Hospital Active not recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 3

DateIssuerRelease
2024-08-22 Ractigen Therapeutics. Regulatory Ractigen Therapeutics Announces U.S. FDA Orphan Drug Designation (ODD) granted to RAG-18 for the treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy ractigen.com ↗
Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-18, one of the company’s lead saRNA product candidates.
2024-07-25 Ractigen Therapeutics. Regulatory Ractigen Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to RAG-18 for the treatment of Duchenne Muscular Dystrophy ractigen.com ↗
Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to RAG-18, one of the company’s lead saRNA product candidates for the treatment of Duchenne Muscular Dystrophy (DMD).
2023-11-02 Ractigen Therapeutics. Results Ractigen Presents Preclinical Results of Its saRNA Therapeutic Targeting DMD/BMD at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting ractigen.com ↗
In a proof-of-concept efficacy study in MDX mice bearing the human UTRN promotor, systemic administration of a lead saRNA conjugated to Ractigen’s proprietary LiCO TM delivery system ameliorated muscle damage.

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as RAG-18 ClinicalTrials.gov intervention name — accepted as the source's own label NCT07282652 ↗
Modality Small activating RNA (saRNA) “small activating RNA (saRNA)” NCT07282652 ↗
Route Intravenous “ascending intravenous doses” NCT07282652 ↗