Drug Repurposing Hub

The Drug Repurposing Hub is a curated and annotated collection of compounds developed and maintained by the Broad Institute’s Center for the Development of Therapeutics (CDoT). The Hub represents a powerful resource for drug discovery, enabling researchers to explore known biological targets and pathways, discover new biological insights, and identify new therapeutic applications for existing drugs.

Overview

Drug repurposing—finding new uses for existing drugs—offers significant advantages over traditional drug development. By testing thousands of already-approved or clinically-tested drugs for their ability to treat different diseases, researchers can potentially accelerate therapeutic development while leveraging existing safety and pharmacokinetic data.

The Broad Institute launched the Drug Repurposing Hub in 2015, initially to accelerate cancer treatments. Since then, the resource has expanded to support research across dozens of disease areas, including tuberculosis, kidney disease, infectious diseases, and many others.

Collection Statistics

The Drug Repurposing Hub contains:

• 7,539 unique compounds
• 2,430 protein targets
• 826 drug indications
• 5,506 compounds in the screening library (90% FDA-approved or in clinical trials)
• ~2,400 additional compounds available for follow-up studies

Compound Categories

The collection includes three main categories of compounds:

1. FDA-Approved Drugs: Compounds already approved for clinical use
2. Clinical Trial Drugs: Compounds currently in various phases of clinical development
3. Pre-clinical Tool Compounds: Selected compounds useful for target validation and biological research

All compounds are:

• Curated and annotated with extensive metadata
• Characterized for structure, targets, mechanisms, and indications
• Quality-controlled (>85% purity by UPLC-MS)
• Well-characterized with known biological activities

Key Features

Comprehensive Annotations

Each compound in the Hub is annotated with:

• Chemical structure: Structural information and identifiers
• Targets: Known protein targets and binding interactions
• Mechanisms of action: How the compounds work at the molecular level
• Indications: Diseases and conditions the compounds are used to treat
• Clinical development phase: FDA approval status or clinical trial phase
• Therapeutic areas: Disease areas and biological systems

Screening Library Access

The physical library is available for screening through two formats:

1. Full Library: 5,506 compounds at single concentration as assay-ready plates
2. REPO1 Subset: Multi-dose format with 5-point, 10-fold dilution series

Libraries are available to:

• Academic and research institutions
• Non-profit organizations worldwide
• Both on-site at Broad Institute and remote locations
• On a cost-recovery basis for non-profit institutions

Note: Individual compounds are not sold separately. A small subset of controlled substances is only available for screening at the Broad Institute.

Data Portal

The interactive data portal allows users to:

• Search compounds by name, structure, target, or indication
• Explore relationships between compounds, targets, and diseases
• Filter by clinical development phase, therapeutic area, or mechanism
• Access downloadable batch data files

Integration with Other Projects

The Drug Repurposing Hub compounds are profiled by multiple complementary projects:

• Connectivity Map (CMap): Gene expression profiles
• Cancer Dependency Map/PRISM: Cancer cell line vulnerability data
• NIH LINCS: Library of Integrated Network-Based Cellular Signatures
• JUMP Cell Painting Consortium: Morphological profiling

Evolution of the Hub

2015: Initial launch with 4,707 compounds at single dose Expansion: Grew to 6,801 compounds with multi-dose formats through philanthropic support Current: 5,506-compound screening library plus ~2,400 follow-up compounds

The expansion enabled:

• Significant data informatics buildout
• Increased compound stock for multiple replatings
• ML-enabled dataset creation through curated metadata
• Discovery of novel connections across diseases, cell lines, and compounds

Applications

The Drug Repurposing Hub supports multiple research approaches:

Target-Based Discovery

• Explore compounds with known activities against specific biological targets
• Understand pathways and biological mechanisms
• Validate new therapeutic targets

Phenotypic Screening

• Screen compounds in disease-relevant cellular assays
• Identify new indications when disease mechanisms are poorly understood
• Discover unexpected biological activities

Biological Insights

• Pair experimental screening results with curated metadata
• Generate ML-enabled datasets for computational analysis
• Uncover disease characteristics and mechanisms

Data Availability and Licensing

• Metadata Access: Freely available for research use without registration
• License: Compound metadata available under CC-BY 4.0 license
• Usage: Generated for research purposes only
• Restrictions: Cannot be repackaged or redistributed for commercial purposes without permission
• Clinical Use: Not intended for clinical treatment or commercial marketing

Note: Downstream experimental data using the library may have different license terms.

Collaboration Opportunities

The Broad Institute collaborates with many groups for screening:

• Academic institutions
• Research organizations
• Non-profit entities

The Center for the Development of Therapeutics (CDoT) offers:

• Decades of screening experience
• Cost-effective screening services
• Wide range of cellular assays
• Expert support for assay development and execution

Contact: repurposing@broadinstitute.org

Compound Submission

Researchers can contribute to the collection by:

1. Reviewing the Compound Intake Policy
2. Submitting compounds through the online form
3. Working with the team to integrate valuable compounds

The original collection was made possible through philanthropic support, and the Hub continues to grow through community contributions.

Quality Control

All small molecule compounds in the library are:

• Confirmed for >85% purity by UPLC-MS at time of addition
• Stored under appropriate conditions
• Tracked for stability and quality over time

Citation

If you use the Drug Repurposing Hub in your research, please cite:

Corsello SM, Bittker JA, Liu Z, Gould J, McCarren P, Hirschman JE, Johnston SE, Vrcic A, Wong B, Khan M, Asiedu J, Narayan R, Mader CC, Subramanian A, Golub TR (2017). “The Drug Repurposing Hub: a next-generation drug library and information resource.” Nature Medicine, 23:405–408.

Team

The Drug Repurposing Hub is developed and maintained by:

• Steven Corsello - Founder of REPO Hub
• Todd Golub - Core Institute Member
• Sandy Gould - Senior Director, Head of Drug Discovery, CDoT
• And dedicated team members from CDoT

Acknowledgements

Funding support from:

• Anonymous donor (primary support for collection subsidy)
• NIH LINCS Program (grant 3U54 HG006093)
• NIH BD2K Program (grant 5U01HG008699)
• NIH training grant T32 CA009172
• NIH/Harvard Catalyst training award KL2 TR001100
• Conquer Cancer Foundation of ASCO Young Investigator Award

Special thanks to curators of public drug databases, chemical vendors, and assay teams.

Contact

For screening inquiries, compound submissions, or general information:

• Email: repurposing@broadinstitute.org
• Website: https://repo-hub.broadinstitute.org/repurposing

Developed by: Center for the Development of Therapeutics (CDoT)
Institution: Broad Institute of MIT and Harvard