Drug intelligence / Profile preview

scFvαIIbβ3 mRNA

Development stage
Preclinical
Lead developer
Baker Heart and Diabetes Institute
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

scFvαIIbβ3 mRNA is an investigational mRNA-based therapeutic encoding a conformation-specific single-chain variable fragment (scFvαIIbβ3) that selectively targets the activated platelet integrin GPIIb/IIIa (integrin αIIbβ3). Developed by the Baker Heart and Diabetes Institute, this therapeutic is designed for safer thromboprophylaxis. By selectively binding to the activated conformation of GPIIb/IIIa, the expressed scFv inhibits pathological thrombosis while preserving physiological hemostasis, thereby mitigating the bleeding risks associated with conventional antiplatelet therapies. The mRNA is formulated in a novel lipid nanoparticle system (OryKL) composed of γ-oryzanol and DLin-KC2-DMA for systemic delivery. Preclinical studies have demonstrated that both conventional mRNA and self-amplifying mRNA (samRNA) constructs provide durable antithrombotic protection in vivo without prolonging bleeding times or causing systemic toxicity.

Other names
platelet-targeted self-amplifying mRNAscFvαIIbβ3 samRNAscFvαIIbβ3 self-amplifying mRNAscFvαIIbβ3-encoding mRNA
02

Targets

GPIIb/IIIa (Integrin alpha-IIb/beta-3)

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