Drug intelligence / Profile preview

ORN-101

Development stage
Unknown
Lead developer
Orna Therapeutics
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

ORN-101 is a development-stage, off-the-shelf immunotherapy that uses engineered circular RNA (oRNA) delivered via a proprietary lipid nanoparticle (LNP) to generate chimeric antigen receptor (CAR)-expressing immune cells directly within the patient. This *in situ* CAR approach enables the body’s own immune cells to be reprogrammed to target and eradicate cancer cells expressing CD19, without the need for ex vivo cell manipulation or lymphodepletion. The therapy is designed for redosability and dose control, aiming to overcome limitations of traditional CAR-T therapies while maintaining efficacy. Preclinical studies have demonstrated potent tumor eradication at significantly lower doses compared to earlier versions. Developed by Orna Therapeutics, this novel modality leverages optimized IRES elements for high protein expression and durable therapeutic effect[1][2][4][8].

02

Targets

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