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OTX-101 (Orion Therapeutics)
- Development stage
- Preclinical
- Lead developer
- Ocular Therapeutix
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravascular
01
Overview
OTX-101 is an investigational siRNA-based therapeutic being developed by Orion Therapeutics for the treatment of vascular conditions such as restenosis and intimal hyperplasia. It utilizes the proprietary GENESYS™ lipid nanoparticle (LNP) delivery platform, which is engineered to target the exposed subendothelial matrix of injured vascular tissues, allowing for localized delivery to the site of vascular injury. The siRNA payload is designed to inhibit enzymes that drive the proliferation of vascular smooth muscle cells, thereby preventing the pathological vessel narrowing that often follows vascular interventions. The program is currently in the IND-enabling stage, with support from the National Science Foundation (NSF) Phase II STTR program and a projected development timeline extending into 2027-2028.
02
Targets
CN (Calcineurin)
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