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CRISPR-like NUC-LNP

Development stage: Preclinical
Lead developer: InhaTarget Therapeutics
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration: Inhalation

Overview

CRISPR-like NUC-LNP is an inhaled genetic therapy in research-stage development by InhaTarget Therapeutics for the treatment of lung fibrosis and lung cancer. The therapeutic utilizes a gene-editing-like nuclease approach delivered via InhaTarget's proprietary lipid nanoparticle (LNP) formulation platform, which is designed to target lung macrophages. By delivering the CRISPR-like nuclease directly to the lungs via inhalation, the therapy aims to achieve localized therapeutic effects while minimizing systemic exposure and toxicity.

Targets

NFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)

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