Drug intelligence / Profile preview

BB-501

Development stage
Preclinical
Lead developer
Benitec Biopharma
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral
01

Overview

**BB-501** is an investigational ddRNAi (DNA-directed RNA interference) therapeutic developed by Benitec Biopharma as a follow-on to BB-401, designed to silence **EGFR** (epidermal growth factor receptor) expression for the treatment of **head and neck cancer**. It functions by delivering a DNA plasmid that expresses short hairpin RNAs to induce potent, durable gene knockdown of EGFR, a key driver of tumor growth in squamous cell carcinomas of the head and neck. Preclinical studies in mouse xenograft models have demonstrated anti-tumor efficacy, and clinical data from the related BB-401 program are informing its development pathway, with current global R&D status listed as **Pending**.[1][3][5][8][16]

02

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