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A phase 1/2 randomized, double-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study of RAG-17, an siRNA therapy targeting SOD1-mutated amyotrophic lateral sclerosis (ALS)

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Trial Profile

A phase 1/2 randomized, double-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study of RAG-17, an siRNA therapy targeting SOD1-mutated amyotrophic lateral sclerosis (ALS)

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 12 Feb 2026

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At a glance

  • Drugs RAG-17 (Primary)
  • Indications Amyotrophic lateral sclerosis
  • Focus Adverse reactions; Therapeutic Use

Most Recent Events

  • 12 Feb 2026 New trial record
  • 13 Jan 2026 According to Ractigen Therapeutics media release, the participating sites include Beijing Tiantan Hospital, Capital Medical University (led by Dr. Yi-Long Wang), The Second Affiliated Hospital, Zhejiang University School of Medicine (led by Dr. Zhi-Ying Wu), West China Hospital of Sichuan University (led by Dr. Hui-Fang Shang), Fujian Medical University Union Hospital (led by Dr. Zhan-Yu Zou) and the First Affiliated Hospital, Sun Yat-sen University (led by Dr. Jing-Sheng Zeng) .
  • 13 Jan 2026 According to Ractigen Therapeutics media release, after the successful completion of the Phase I single ascending dose (SAD) portion of this study, the 1st patient has been dosed in the Phase II part of this trial, under the leadership of Dr. Zhi-Ying Wu, head of the Department of Medical Genetics/Center for Rare Diseases.

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