Target intelligence / Profile preview

Targeted delivery of active substances

Molecular classification
Other
01

Overview

Targeted delivery of active substances is a pharmacological strategy designed to increase the concentration of a therapeutic agent in a specific body region, tissue, or cell type while minimizing its presence in non-target areas [1]. This concept does not represent a discrete biological target like a receptor or enzyme; rather, it is a technological framework encompassing various delivery platforms such as antibody-drug conjugates (ADCs), nanoparticles, and liposomes [2][3]. The goal is to enhance the therapeutic index of drugs by improving efficacy and reducing systemic side effects, which is particularly critical for highly potent or toxic agents [4]. Many targeted systems utilize ligands that recognize specific biomarkers on the surface of diseased cells or exploit unique environmental conditions, such as the acidic pH of a tumor or the presence of specific enzymes, to trigger drug release [5]. In modern medicine, this approach is a cornerstone of oncology, allowing for the precise delivery of cytotoxic payloads to malignant cells while sparing healthy tissue [6].

Other names
Targeted drug deliverySite-specific drug deliveryDrug delivery systems (DDS)Smart drug deliveryTargeted therapy delivery
02

Mechanism of action

Improving the therapeutic index by using carriers (e.g., antibodies, nanoparticles, liposomes) or chemical modifications to localize drug concentration at specific physiological sites, thereby enhancing efficacy and reducing systemic toxicity [1][2].

03

Biological functions

Other
04

Disease associations

CancerInflammationInfectionCardiovascular diseaseNeurodegenerative disease
05

Safety considerations

Off-target toxicity due to non-specific uptakeImmunogenicity of delivery vehiclesPremature release of payload (leakiness)Complex manufacturing and characterizationCarrier-related toxicity
06

Interacting drugs

Trastuzumab emtansine

4 more in the full profile.

07

Biomarkers

Cell-surface antigens (e.g., HER2, CD19)Tumor microenvironment pHOverexpressed proteasesEnhanced Permeability and Retention (EPR) effect

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