Related Experiment Video
Updated: Feb 22, 2026

05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
10.2K
Uncovering novel repositioning opportunities using the Open Targets platform.
Mugdha Khaladkar1, Gautier Koscielny2, Samiul Hasan3
1GSK, 709 Swedeland Road, King of Prussia, PA 19406, USA.
Drug Discovery Today
|September 19, 2017
Summary
This study uses the Open Targets platform to find new uses for existing drugs. It identifies drug repositioning opportunities by analyzing target-disease associations for novel therapeutic applications.
Area of Science:
- Computational biology
- Pharmacology
- Genomics
Background:
- The Open Targets platform integrates diverse evidence linking drug targets to human diseases.
- Identifying novel therapeutic applications for existing drugs (drug repositioning) is a key strategy in pharmaceutical research.
Purpose of the Study:
- To systematically identify novel drug repositioning opportunities by leveraging integrated data from the Open Targets platform.
- To classify and analyze these opportunities based on evidence strength and therapeutic area.
Main Methods:
- A computational workflow was developed to mine the Open Targets platform for target-disease associations.
- Data mining focused on evidence categories to assess the strength of target-disease pairings.
- Identified opportunities were classified by evidence count, therapy area of origin, and repositioning scope (within or across areas).
Main Results:
- The workflow successfully identified potential drug repositioning candidates by analyzing integrated evidence.
- Novel target-disease associations were uncovered, suggesting new therapeutic uses for marketed or investigational drugs.
- Opportunities were categorized to provide a structured overview of repositioning potential.
Conclusions:
- The Open Targets platform is a valuable resource for uncovering drug repositioning opportunities.
- Computational analysis of integrated evidence can efficiently identify novel therapeutic strategies.
- This approach facilitates the exploration of new uses for drugs in clinical development or on the market.
More Related Videos
Related Concept Videos
Drug Discovery: Overview
12.2K
Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
12.2K
Pharmacogenomics: Identification of New Drug Targets
29
Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
29
Targets for Drug Action: Overview
10.7K
Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
10.7K
Structure-Activity Relationships and Drug Design
1.9K
Drug design is a dynamic field that involves discovering and developing new medications based on specific biological targets. This process heavily relies on structure-activity relationships (SAR) and quantitative structure-activity relationships (QSAR) to guide the design and optimization of efficient drugs.
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence...
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence...
1.9K
Modified-Release Drug Delivery Systems: Site-Targeted
33
Site-targeted drug delivery systems enhance therapeutic efficacy while minimizing systemic toxicity and treatment costs. Unlike conventional methods, these systems ensure precise drug delivery, improving bioavailability and reducing side effects. Targeted drug delivery is classified into three levels. First-order targeting directs drugs to the capillary beds of specific organs or tissues. Second-order targets specific cell types, such as tumor cells, using receptor-mediated interactions.
33
Prodrugs
4.5K
Prodrugs are a class of pharmaceutical compounds that undergo a biotransformation process within the body to be converted into a pharmacologically active drug. Prodrugs are designed to improve the therapeutic properties of the parent drug, such as enhancing bioavailability, increasing stability, or reducing toxicity. The concept of prodrugs revolves around modifying the chemical structure of the original drug to make it more effective or convenient for administration.
Prodrugs help overcome...
Prodrugs help overcome...
4.5K

