Drug intelligence / Profile preview

CEQ501

Development stage
Discontinued
Lead developer
Marina Biotech
Modality
Defined Consortia → Multi-strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Microbial Metabolites → Microbiome-Derived Products → Microbiome Therapeutics, Synthetic Biology Platforms → Engineered Microbial Therapeutics → Microbiome Therapeutics, Fresh FMT → Fecal Microbiota Transplantation (FMT) → Microbiome Therapeutics, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Genetically Modified Bacteria → Engineered Microbial Therapeutics → Microbiome Therapeutics, Yeast Strains → Single Strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Frozen FMT → Fecal Microbiota Transplantation (FMT) → Microbiome Therapeutics, Bacterial Strains → Single Strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Microbial Proteins → Microbiome-Derived Products → Microbiome Therapeutics, RNA Therapeutics → Nucleic Acid Therapeutics, Complex Communities → Multi-strain Products → Live Biotherapeutic Products → Microbiome Therapeutics
Administration
Oral
01

Overview

CEQ501 is an experimental oral RNA interference (RNAi) therapeutic developed for the treatment of Familial Adenomatous Polyposis (FAP), a hereditary condition characterized by the development of numerous precancerous polyps in the colon. Developed by Cequent Pharmaceuticals, the drug utilizes a proprietary TransKingdom RNAi (tkRNAi) platform. This technology employs engineered, non-pathogenic *Escherichia coli* as a delivery vehicle to transport short hairpin RNA (shRNA) directly into intestinal epithelial cells. The shRNA is designed to silence the *CTNNB1* gene, which encodes beta-catenin, a protein whose overexpression drives polyp formation in FAP. Following the acquisition of Cequent by MDRNA (later Marina Biotech) in 2010, CEQ501 served as the lead candidate before being succeeded by CEQ508, a refined version of the same therapeutic approach.

02

Targets

CTNNB1 (Beta-catenin)

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