CMTA Strategy To Accelerate Research (CMTA-STAR) Alliance Partner Vanda Pharmaceuticals has dosed the first patient in an early phase clinical trial of VCA-894A, an investigational 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.
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.
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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Not yet registered with Patients as Partners in Research, but want to be ready if opportunities like Vanda’s CMT2S clinical trial begins recruiting? Register today to ensure you are prepared. Registration is free, secure, and open to all individuals with CMT. You will receive updates on CMT research and be positioned to participate if and when future studies like Vanda’s begin recruiting.
