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Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the first dose in the first-in-human clinical trial to evaluate the safety and tolerability of VCA-894A, an antisense oligonucleotide (ASO) therapeutic, for a patient with a rare variant in the IGHMBP2 gene causing Charcot-Marie-Tooth disease Type 2S (CMT2S).
VCA-894A is being developed for a patient who was first diagnosed at the age of 5 with a rare subtype of CMT disease known as CMT2S.1 CMT2S is an inherited neuromuscular disorder that progressively leads to muscle weakness and loss of motor function, and has an estimated prevalence of less than 1 in 1,000,000 worldwide.2 The severity and clinical presentations of CMT2S are influenced by the diverse genetic variants associated with CMT disease.
VCA-894A targets a patient-specific IGHMBP2 variant and has been previously shown to restore expression levels of the IGHMBP2 gene in an "organ-on-a-chip" NMJ (neuromuscular junction) system derived from the patient's cells, highlighting the exciting potential of this approach for personalized therapeutics for rare diseases. This platform has the potential to unlock the development of treatments that can address significantly unmet medical needs based on identified causative genetic variants.
Posted In: VNDA
