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CEQ501
- Development stage
- Discontinued
- Lead developer
- Biodexa Pharmaceuticals
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Bacterial Strains → Single Strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Yeast Strains → Single Strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Defined Consortia → Multi-strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Complex Communities → Multi-strain Products → Live Biotherapeutic Products → Microbiome Therapeutics, Fresh FMT → Fecal Microbiota Transplantation (FMT) → Microbiome Therapeutics, Frozen FMT → Fecal Microbiota Transplantation (FMT) → Microbiome Therapeutics, Genetically Modified Bacteria → Engineered Microbial Therapeutics → Microbiome Therapeutics, Synthetic Biology Platforms → Engineered Microbial Therapeutics → Microbiome Therapeutics, Microbial Metabolites → Microbiome-Derived Products → Microbiome Therapeutics, Microbial Proteins → Microbiome-Derived Products → Microbiome Therapeutics, RNA Therapeutics → Nucleic Acid 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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