Our Science

We start by thinking differently about research and development (R&D). Leading with science and compassion, our team focuses on improving patients’ lives by maximizing the potential of existing medicines as well as developing new life-changing treatments. It’s our willingness to take calculated risks that deliver life-altering medicines for underserved patients.

A Shared Team Vision

Our combined expertise and agile approach to R&D make us unique. With an entrepreneurial mindset that unites collaboration and innovation, our R&D, technical operations, medical affairs and commercial teams work seamlessly together to make quick decisions throughout all stages of development. Our team uses extensive research experience to target, design and execute R&D programs that deliver profoundly impactful medicines to underserved patients.

Our Approach

New Indications



Clinical Trials

New Business

Horizon R&D creatively combines scientific excellence with our compassion for patients to develop novel medicines, identify potential assets, design programs with a high likelihood of success and execute them efficiently.

We know innovation doesn’t just occur within our walls. We deliver in new ways by building partnerships that nurture innovation and maximize the therapeutic potential of our pipeline. We cultivate relationships with patients as well as industry partners, patient advocacy groups and medical specialists, turning our partners’ most promising ideas into breakthrough treatments for patients.

Delivering New Hope for Patients

Rare Diseases

Rare diseases pose a significant and unique challenge in medicine and care to small populations of patients. With only 5 percent of the 7,000 known rare diseases having approved treatments1, the insights we gain from the rare disease community are essential to helping us focus our development process on the most critical of patient needs.

Rheumatic Diseases

Rheumatic diseases affect the joints, tendons, ligaments, bones and muscles, causing substantial, chronic pain and reduced mobility. These diseases, including uncontrolled gout, often present significant challenges to diagnosis and treatment.

Our R&D Footprint

Our R&D team, spanning our South San Francisco, Deerfield, Gaithersburg, Rockville and Dublin offices, applies scientific expertise and courage to maximize the potential of medicines under our care to their fullest potential. We promote a philosophy of getting the job done while staying true to the company’s mission, values and culture. Our South San Francisco facility is 20,000 square feet, including laboratory space that is equipped for bioanalytical method development, pre-manufacturing formulation and process development.

1 Miyamoto BE, Kakkis ED. The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases. Orphanet Journal of Rare Diseases. 2011;6(1):49. doi:10.1186/1750-1172-6-49.