Ractigen Therapeutics has dosed the first subject in the randomised Phase I clinical trial of its small interfering RNA (siRNA) therapy, RAG-17, for treating amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene.
The patient was dosed at Zhejiang University School of Medicine’s Second Affiliated Hospital.
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The placebo-controlled, double-blind trial aims to assess the pharmacokinetics, preliminary efficacy, pharmacodynamics, and safety/tolerability of the therapy in individuals with SOD-ALS.
It is being led by Dr Yilong Wang from Beijing Tiantan Hospital of Capital Medical University, Dr Zhiying Wu from the Second Affiliated Hospital, along with Dr Huifang Shang from West China Hospital of Sichuan University.
Ractigen Therapeutics founder and CEO Dr Long-Cheng Li said: “The first patient dosed in the RAG-17 trial marks a pivotal milestone in our mission to combat ALS, one of the most devastating neurodegenerative diseases.
“This achievement underscores our unwavering commitment to advancing RNA-based therapies that have the potential to transform the lives of patients and families affected by rare and severe conditions like ALS.”
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By GlobalDataRAG-17 as an ALS therapy was further supported by ‘promising’ clinical data from an Investigator-Initiated Trial (IIT) conducted last month. These outcomes indicated that the intrathecally administered therapy was well-tolerated at all dose levels, with only mild adverse events reported.
Leveraging the company’s Smart Chemistry Aided Delivery (SCAD) platform, RAG-17 is tailored for the SOD1 gene suppression in individuals with this neurodegenerative condition having pathogenic mutations. The platform is specifically intended for the delivery of duplex RNA, such as siRNA, into the central nervous system (CNS) via intrathecal injection.
The therapy also improved motor function and extended survival as per the preclinical studies along with those using the hSOD1G93A mouse model.
In March last year, the US Food and Drug Administration (FDA) granted the therapy an orphan drug designation (ODD), followed by clearance of its investigational new drug (IND) application.
China National Medical Products Administration (NMPA)’s Center for Drug Evaluation (CDE) also approved the IND in May this year.
