Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
LNA-224
- Development stage
- Preclinical
- Lead developer
- Icahn School of Medicine
- 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
- Inhalation
01
Overview
LNA-224 is a chemically modified antisense oligonucleotide (specifically a locked nucleic acid, or LNA) designed to target and inhibit microRNA-224-5p (miR-224). It was initially developed by the Icahn School of Medicine at Mount Sinai and is being investigated for the treatment of pulmonary arterial hypertension (PAH). In preclinical models of PAH (such as Sugen/Hypoxia and monocrotaline rodent models), intratracheal delivery of aerosolized LNA-224 has been shown to significantly attenuate disease severity, reduce pulmonary artery and right ventricular pressure, and improve right ventricular function. Mechanistically, miR-224 targets multiple components of the bone morphogenetic protein (BMP) and transforming growth factor-beta (TGF-β) signaling pathways; inhibiting miR-224 with LNA-224 restores the balance between growth-inhibitory BMP signaling and growth-promoting TGF-β signaling in pulmonary arterial smooth muscle cells (PASMCs).
- Other names
- LNA-miR-224LNA-miR224LNA-miR 224locked nucleic acid targeting miR-224locked nucleic acid targeting microRNA-224
Beyond the preview
Go deeper on LNA-224.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect 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 LNA-224.