Top 5 Companies Leading Drug Repurposing Efforts in the U.S.

Published On : Feb 17, 2025

Drug repurposing, or drug repositioning, has gained significant momentum in the U.S. pharmaceutical industry as companies seek faster and cost-effective ways to develop treatments for various diseases. The traditional drug discovery process is time-consuming and expensive, often taking over a decade and billions of dollars to bring a new drug to market.

By contrast, repurposing existing drugs-those that have already been approved for other conditions-can dramatically reduce the time and cost involved in drug development. The COVID-19 pandemic further highlighted the importance of drug repurposing, as researchers scrambled to identify existing medications that could be used to treat the virus, leading to emergency use authorizations for drugs such as remdesivir and dexamethasone.

Top 5 Companies Working to Repurpose Drugs in the U.S.

Top 5 Key Innovators in Drug Repurposing Field in the U.S.

1. Recursion Pharmaceuticals

Recursion Pharmaceuticals, headquartered in Salt Lake City, Utah, integrates novel technologies across biology, chemistry, automation, data science, and engineering to map and decode human biology. The company utilizes high-throughput screening and machine learning algorithms to analyze vast datasets, identifying potential new uses for existing compounds. This approach enables Recursion to systematically explore drug repurposing opportunities across various disease areas.

To stay competitive, Recursion has established strategic partnerships and collaborations. Notably, in 2020, the company entered into a strategic collaboration with Bayer to discover and develop treatments for fibrotic diseases. In 2023, they extended this partnership to focus on oncology research. Additionally, Recursion has acquired companies in the AI-enabled drug discovery space, such as Cyclica and Valence, to bolster its capabilities. These initiatives enhance Recursion's technological infrastructure and extend its pipeline of repurposed drug candidates.

2. Melior Discovery

Melior Discovery, based in Exton, Pennsylvania, specializes in drug repositioning through its proprietary phenotypic screening platform. This platform allows the company to identify new therapeutic indications for existing drugs by observing their effects across various biological models. Melior also offers contract research services, providing animal models representing different disease conditions to support drug discovery efforts.

To maintain competitiveness, Melior has formed partnerships with major pharmaceutical companies, including Pfizer, Merck & Co., Johnson & Johnson, and AstraZeneca. These collaborations leverage Melior's drug repositioning technology to explore new therapeutic uses for existing compounds. Additionally, Melior has utilized its platform to discover its own drug candidates, bolstering them into clinical development.

3. United Therapeutics

United Therapeutics, headquartered in Silver Spring, Maryland, focuses on developing innovative therapies for pulmonary arterial hypertension (PAH) and other serious conditions. The company has a history of repurposing existing compounds to address unmet medical needs. For instance, United Therapeutics developed Remodulin, a prostacyclin vasodilator, as an alternative treatment for PAH patients.

To stay competitive, United Therapeutics has pursued strategic acquisitions and collaborations. The company acquired Revivicor, a company focused on developing genetic biotechnology platforms, to explore alternative tissue sources for treatment. Additionally, United Therapeutics has entered into licensing agreements and collaborations with companies such as Eli Lilly and Company, DEKA Research & Development, and MannKind Corporation to extend its therapeutic offerings.

4. Vertex Pharmaceuticals

Vertex Pharmaceuticals, based in Boston, Massachusetts, is renowned for its work in genetic therapies, particularly for cystic fibrosis (CF). The company has engaged in drug repurposing by exploring new therapeutic applications for its existing compounds. Vertex has entered into collaborations to develop gene-editing therapies for genetic diseases, aiming to broaden the potential uses of its compounds beyond their original indications.

To maintain a competitive edge, Vertex has established research collaborations with companies such as CRISPR Therapeutics and Moderna to develop new therapies. These partnerships aim to leverage existing compounds and technologies to address a broader range of diseases. By exploring new applications for its compounds, Vertex seeks to maximize the therapeutic potential of its drug portfolio.

5. Ginkgo Bioworks

Ginkgo Bioworks, headquartered in Boston, Massachusetts, utilizes artificial intelligence platforms and synthetic biology to engineer enzymes, proteins, and drug molecules. The company employs AI-based tools to identify new therapeutic uses for existing compounds, facilitating drug repurposing efforts. Ginkgo's platform combines computational design with high-throughput genetic engineering and screening to discover novel applications for existing drugs.

Ginkgo's product line includes a range of engineered organisms and biological products designed for various industries, including pharmaceuticals. The company's platform has been utilized to develop diagnostics, optimize and manufacture medicines and vaccines, and support diverse pandemic responses, such as during the COVID-19 pandemic. By leveraging its synthetic biology platform, Ginkgo aims to accelerate the development and production of essential medicines, contributing to more efficient drug repurposing efforts.

Companies to Focus on Precision Medicine and Personalized Treatments in Future

The future of drug repurposing in the U.S. looks promising, driven by continued developments in AI, regulatory support, and collaborations between pharmaceutical companies, biotech firms, and research institutions. Growing emphasis on precision medicine and personalized treatments is likely to further extend opportunities for drug repurposing.

Companies are increasingly recognizing the value of repurposing as a strategy to diversify their pipelines and address pressing medical needs. As more success stories emerge, the pharmaceutical industry is set to allocate greater resources toward systematic drug repurposing efforts, ensuring faster and more cost-effective treatment options for a wide range of diseases.

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