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eAFP-VISA-BikDD

Development stage: Preclinical
Lead developer: University of Texas MD Anderson Cancer Center
Modality: Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Gene Therapies
Administration: Intravenous

Overview

**eAFP-VISA-BikDD** is a non-viral, liposome-encapsulated gene therapy designed for the targeted treatment of hepatocellular carcinoma (HCC). The construct centers on a modified pro-apoptotic Bik gene (BikDD) under the control of an α-fetoprotein enhancer/promoter (eAFP) for liver cancer specificity, and a VISA (VP16-GAL4-WPRE integrated systemic amplifier) platform that enhances transgene expression. Systemic delivery of eAFP-VISA-BikDD induces apoptosis selectively in HCC cells via the mitochondrial pathway, activating Bax and caspase-3, and effecting cytochrome c release. Preclinical studies show potent antitumor activity, especially when combined with doxorubicin, and a favorable safety profile in animal models. The primary indication is advanced hepatocellular carcinoma[2].

Other names: eAFP-VISA-BikDD

Targets

BAKBCL2L1 (B-cell lymphoma-extra large protein)MCL1 (Myeloid cell leukemia sequence 1)BAX (Apoptosis regulator BAX)

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