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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
01
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.
- Other names
- VCA-894VCA894VCA 894antisense oligonucleotide targeting IGHMBP2 cryptic splice site
02
Targets
IGHMBP2 (Immunoglobulin mu-binding protein 2)
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