Drug intelligence / Profile preview

RePair53

Development stage
Preclinical
Lead developer
Sungkyunkwan University
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody-Based Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intratumoral
01

Overview

**RePair53** is a preclinical targeted RNA therapeutic platform for **p53-mutant cancers**. It co-delivers a pan-TP53 small interfering RNA designed to suppress endogenous mutant p53 transcripts and a codon-optimized wild-type p53 mRNA engineered to evade siRNA degradation, thereby restoring functional p53 protein expression. The RNA payloads are co-encapsulated in lipid nanoparticles functionalized with a panitumumab-ApoA1 fusion targeting moiety for epidermal growth factor receptor-directed tumor delivery. In a CAL-27 xenograft model, RePair53 produced greater antitumor activity than p53 mRNA alone.

Other names
Delete-and-Replace platformRestoration of p53 via Paired interference and replacement
02

Targets

TP53 (Cellular Tumor Antigen p53 R175H)Mutant Tumor Protein p53 messenger RNA

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