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ION356
- Development stage
- Phase 1
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Gene Therapies, 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
ION356 is an investigational antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of Pelizaeus-Merzbacher disease (PMD), a rare, severe leukodystrophy caused by duplications or mutations in the PLP1 gene. The drug is designed to silence or inhibit expression of the PLP1 gene, thereby reducing levels of proteolipid protein 1 and addressing the underlying cause of PMD. It is administered via intrathecal injection and has received Orphan Drug Designation and Rare Pediatric Disease Designation from regulatory authorities. Clinical trials are ongoing to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in pediatric patients with classic PMD[2][4][5][6][7][9][10].
- Other names
- ION-356ION356ION 356
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