Drug intelligence / Profile preview

ad-her2-sirna

Development stage
Preclinical
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

ad-her2-sirna is an investigational RNA-based therapeutic designed to silence the expression of the human epidermal growth factor receptor 2 (HER2, also known as ErbB2) using small interfering RNA (siRNA) technology. HER2 is a transmembrane receptor tyrosine kinase that is overexpressed in approximately 20% of breast cancers, often conferring aggressive tumor characteristics and resistance to existing HER2-targeted therapies like trastuzumab and lapatinib. ad-her2-sirna consists of a synthetic siRNA sequence targeting HER2 mRNA, delivered systemically, frequently via nanoparticle-based delivery platforms. In preclinical studies, HER2 siRNA—including variants like siHER2 d75—has been shown to potently reduce HER2 protein levels, induce apoptotic responses, and inhibit proliferation in both wild-type and drug-resistant HER2-positive breast cancer cell lines, as well as in resistant tumor xenograft models. The siRNA can also silence oncogenic HER2 splice variants implicated in therapy resistance, and extended treatment in cell models has not resulted in resistance development.

02

Targets

ERBB2 (Erb-b2 receptor tyrosine kinase 2)

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