Open Targets Platform: supporting systematic drug–target identification and prioritisation

TLDR

The Open Targets Platform is an open‑source, publicly available knowledgebase and interface that supports systematic target identification and prioritisation for drug discovery, addressing the challenge of developing effective and safe drugs. This article presents recent enhancements to the Open Targets Platform designed to improve target identification and prioritisation for drug discovery. Since the last update, the platform has released ten updates, incorporated evidence from 20 data sources—including CRISPR knockout screens and GWAS—refined its scoring framework, added adverse‑drug‑reaction and tractability assessments, and improved performance and usability.

Abstract

The Open Targets Platform (https://www.targetvalidation.org/) provides users with a queryable knowledgebase and user interface to aid systematic target identification and prioritisation for drug discovery based upon underlying evidence. It is publicly available and the underlying code is open source. Since our last update two years ago, we have had 10 releases to maintain and continuously improve evidence for target-disease relationships from 20 different data sources. In addition, we have integrated new evidence from key datasets, including prioritised targets identified from genome-wide CRISPR knockout screens in 300 cancer models (Project Score), and GWAS/UK BioBank statistical genetic analysis evidence from the Open Targets Genetics Portal. We have evolved our evidence scoring framework to improve target identification. To aid the prioritisation of targets and inform on the potential impact of modulating a given target, we have added evaluation of post-marketing adverse drug reactions and new curated information on target tractability and safety. We have also developed the user interface and backend technologies to improve performance and usability. In this article, we describe the latest enhancements to the Platform, to address the fundamental challenge that developing effective and safe drugs is difficult and expensive.