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VCA-894A (Vanda Pharmaceuticals Inc.)
- Development stage
- Unknown
- 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) therapy developed by Vanda Pharmaceuticals for the treatment of Charcot-Marie-Tooth disease type 2S (CMT2S). It is a 2'-O-methoxyethyl (MOE) phosphorothioate oligonucleotide sodium salt designed as a personalized, patient-specific (n-of-1) therapeutic. VCA-894A specifically targets a rare, paternally inherited cryptic splice site variant (c.1235+894 C>A) in intron 8 of the immunoglobulin mu-binding protein 2 (IGHMBP2) gene. By binding to this aberrant cryptic splice site, VCA-894A prevents the creation of a new splice acceptor site, thereby correcting the pathogenic splicing event, preventing nonsense-mediated decay, and restoring functional IGHMBP2 protein levels.
- Other names
- VCA-894VCA894VCA 894antisense oligonucleotide targeting IGHMBP2 cryptic splice site
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