CMTA Strategy To Accelerate Research (CMTA-STAR) Alliance Partner Vanda Pharmaceuticals has dosed the first healthy volunteer in a clinical trial of its investigational drug VCA 894A, an antisense oligonucleotide (ASO) the company designed to treat CMT2S. This milestone marks early progress toward a potential nucleic acid therapy (NAT) for individuals with this ultra-rare form of CMT.
CMT2S is caused by specific recessive mutations in the IGHMBP2 gene. In preclinical studies, the company reported that VCA 894A was shown to restore IGHMBP2 gene expression in patient-derived neuromuscular junction models. This early phase of the clinical trial will assess safety, tolerability, and early signs of biological activity in participants with CMT2S.
The CMTA-STAR Alliance supports the development of treatments for all types of CMT, including rare subtypes like CMT2S. We are proud to partner with CMTA-STAR Alliance Partner Vanda Pharmaceuticals as they advance this precision-driven program.
CMTA Strategy to Accelerate Research (CMTA-STAR) Alliance Partner Vanda Pharmaceuticals has dosed the first patient in a clinical trial of VCA 894A, an antisense oligonucleotide (ASO) designed to treat Charcot-Marie-Tooth disease type 2S (CMT2S). This milestone marks early progress toward a potential genetic therapy for individuals with this ultra-rare form of CMT. CMT2S is caused by specific recessive mutations in the IGHMBP2 gene. In preclinical studies, the company reported that VCA 894A was shown to restore IGHMBP2 gene expression in patient-derived neuromuscular junction models.
CMTA continues to follow this study with great interest and proudly supports our CMTA-STAR Alliance Partner Vanda Pharmaceuticals as they move VCA-894A into a CMT clinical trial.
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Published on: June 19, 2025