Vanda Pharmaceuticals

Vanda is a leading global biopharmaceutical company developing important new medicines to improve the lives of patients.

Vanda is developing novel ASOs to treat CMT2S driven by IGHMBP2 variants and have programs in both the preclinical and clinical space. Charcot-Marie-Tooth disease Type 2S (CMT2S) may be caused by immunoglobulin mu-binding protein 2 (IGHMBP2) genetic variants. Vanda has developed an antisense oligonucleotide (ASO) therapeutic that targets IGHMBP2 in order to restore proper functioning of the IGHMBP2 protein product. This ASO currently targets a specific CMT2S patient’s IGHMBP2 variant and has been shown to be efficacious in preclinical studies with no adverse events reported. The Investigational New Drug (IND) Application for this ASO has been approved by the Food and Drug Administration (FDA), and the clinical trial for the patient is soon to begin. Vanda Pharmaceuticals is expanding this program and is actively developing an ASO that broadly targets IGHMBP2 so that more patients can be treated.