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avicursen
- Development stage
- Phase 2
- Lead developer
- Percheron Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Avicursen is an antisense oligonucleotide (ASO) designed to reduce inflammation through inhibition of CD49d. It works by reducing CD49d-positive lymphocytes, which could help manage inflammatory diseases. The drug was primarily investigated for non-ambulant Duchenne muscular dystrophy (DMD) but has also been studied for multiple sclerosis and other conditions.
- Other names
- alpha-4 integrin antisense oligonucleotidealpha4 integrin antisense oligonucleotidealpha 4 integrin antisense oligonucleotideCD49d antisense oligonucleotideCD-49d antisense oligonucleotideCD 49d antisense oligonucleotide
02
Targets
ITGA4 (Integrin alpha-4)
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