Our Science

At Horizon, we think differently about research and development (R&D). Our willingness to take calculated risks allows us to deliver new medicines for patients with rare, autoimmune and severe inflammatory diseases.

We apply extensive research experience to target, design and execute R&D programs to deliver medicines that will have a profound impact on underserved patients who don’t have many, if any, options.

Our approach allows us to develop breakthrough treatments and maximize the potential of medicines under our care.

A Shared Team Vision

Our diverse expertise and agility makes us unique. We operate with an entrepreneurial mindset that unites collaboration and innovation. Horizon’s scientists work seamlessly and quickly with colleagues in technical operations, medical affairs and commercial to make decisions with patients in mind, across all stages of development.

Our Approach

New Indications



Clinical Trials

New Business

Collaboration Elevates Our Performance

We know innovation doesn’t just occur within our walls. Horizon builds partnerships that maximize the therapeutic potential of our pipeline. We also cultivate relationships with industry partners, patient advocacy groups and medical specialists, turning our partners’ most promising ideas into breakthrough treatments for patients.

Partnering With Horizon

Delivering New Hope for Patients

Rare Diseases

Rare diseases are disorders that affect a small percentage of the population. There are many different causes of rare disease, with the majority having a genetic link, often triggered by changes in genes or chromosomes. With only five percent of the 7,000 known rare diseases with 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.

Autoimmune Diseases

Autoimmune diseases are the result of an individual’s immune system attacking its own healthy cells and tissue. These diseases often involve autoreactive immune cells that may create autoantibodies, and can result in recurring bouts of disease flares and other complications.

Severe Inflammatory Diseases

Severe inflammatory diseases are characterized by inflammation in the body, including the joints, muscles and other bodily systems. This inflammation can cause substantial and chronic pain, reduced mobility and difficulty in daily life. These diseases often present significant challenges to diagnosis and treatment.

Our R&D Footprint

We have state-of-the-art laboratory facilities in Maryland and California, with resources to evaluate, develop and innovate new medicines.

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.