Ractigen Says China IND Approved for siRNA Candidate that Targets ALS

Suzhou Ractigen Therapeutics reported that a China IND was approved for RAG-17 as a therapy for Amyotrophic Lateral Sclerosis (ALS). RAG-17 is a therapeutic siRNA designed to suppress the SOD1 gene in ALS patients with pathogenic mutations. Of the more than 50 genes associated with ALS, SOD1 gene mutations account for approximately 20% of the cases. Using Ractigen's proprietary SCAD™ delivery platform, RAG-17 combines siRNA with an accessory oligonucleotide to improve efficacy in central nervous system tissues. In preclinical studies, RAG-17 showed the ability to improve motor function and prolong survival. More details....

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