Vanda Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate VCA-894A for the treatment of a patient with Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within the IGHMBP2 gene. VCA-894A is a novel antisense oligonucleotide (ASO) with a mechanism of action that specifically targets a Cryptic splice site variant within IGHMBP2, which causes CMT2S. mutations within IGHMBP2 play a pivotal role in the manifestation of CMT2S, likely due to alpha-motor neuron loss, and consequently peripheral nervous system deterioration.

ASOs have the capacity to modulate gene expression, allowing for the personalized treatment of rare diseases. Delivery of ASOs to the central nervous system has been previously successful in several ASO programs, with a broad applicability in addressing a number of neurodegenerative and neuromuscular disorders. VCA-894 A is a 2'-O-methoxyethyl (MOE) phosphorothioate oligonucleotide sodium salt.

VCA-894 a specifically targets a cryptic splice site variant within I GHMBP2, which cause CMT2S. ASOs may have broad applicability in addressing a Number of disorders, from nervous system treatments to systemic treatments.