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Ractigen Granted Orphan Drug Status for Use of Novel siRNA in ALS

publication date: Mar 9, 2023

Ractigen Therapeutics, a Nantong RNAa company, was granted Orphan Drug Designation in the US for its novel siRNA candidate as a therapy for Amyotrophic Lateral Sclerosis (ALS). RAG-17 is designed to target and knockdown the expression of SOD1 in patients with pathogenic mutations known to cause ALS. The siRNA candidate is conjugated to an accessory oligonucleotide, which enables potent activity in CNS tissue, according to Ractigen. In several preclinical studies, RAG-17 showed significantly higher potency in ALS disease models than competing compounds. More details....

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