Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
ION356
- Development stage
- Phase 1
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → 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
Beyond the preview
Go deeper on ION356.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on ION356.