Vanda Pharmaceuticals Inc. has announced the initiation of a first-in-human clinical trial to assess the safety and tolerability of VCA-894A, a novel antisense oligonucleotide $(ASO.AU)$ therapeutic. This trial targets a patient with a rare variant in the IGHMBP2 gene, which leads to Charcot-Marie-Tooth disease Type 2S (CMT2S), a progressive neuromuscular disorder. VCA-894A is designed to address this specific genetic variant and has previously demonstrated the ability to restore IGHMBP2 gene expression in an "organ-on-a-chip" neuromuscular junction system derived from the patient's cells. The study marks a significant step toward personalized therapeutics for rare diseases, although the results of the trial have not yet been presented.
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