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VCA-894A

Development stage: Phase 2
Lead developer: Vanda Pharmaceuticals
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intrathecal

Overview

VCA-894A is an investigational antisense oligonucleotide (ASO) developed by Vanda Pharmaceuticals in collaboration with OliPass Corporation. It is designed to rescue and restore the activity of the IGHMBP2 protein by correcting its splicing. This drug is being studied for the treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S), specifically in individuals with a confirmed IGHMBP2 intronic cryptic splice variant c.1235+894C>A. VCA-894A is administered via intrathecal injection.

Targets

Immunoglobulin mu-binding protein 2 (IGHMBP2) pre-mRNA cryptic splice site variant

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