Unlocking Disease: Amgen’s Omics Approach Advances Targeted Treatments

Despite advances in medicine, key questions remain: Why do some develop diseases while others don’t? Why does a treatment work for one patient but not another? How can we predict which medicines may be most effective before initiating clinical trials?

Amgen is using omics—the large-scale study of biological data—to help answer these questions and understand health and disease. By integrating genomics (genes), transcriptomics (RNA) and proteomics (proteins) research, Amgen aims to redefine drug discovery, accelerate development and deliver more effective targeted treatments to patients faster than ever before.

Using Omics to Drive Drug Discovery

Amgen’s approach to drug discovery has always been rooted in human biology, with the recognition that understanding genetics—how variations in DNA contribute to disease—could transform drug development. But genes tell only part of the story. Deeper insights come with the integration of multiple layers of biological human data.

• Genomics reveals inherited disease risk and helps discover drug targets.
• Transcriptomics shows how genes are activated or suppressed in disease and treatment.
• Proteomics provides dynamic insights into disease progression and treatment effects at the molecular level.

“A central goal is to harness human data at population scale to understand complex disease and to direct the discovery of medicines,” said Jay Bradner, Executive Vice President of Amgen R&D. “Integrating human genetic variation with clinical outcomes provides unprecedented insights into the cause of disease and, importantly, targets which might offer protection.”

From Data to Breakthroughs

Amgen’s subsidiary, deCODE genetics, a global leader in human genomics based in Iceland, has some of the most extensive collaborations with groups worldwide to study omics data. This allows Amgen to discover genetic risk factors for diseases like cardiovascular disorders, cancer, autoimmune and rare diseases. Integrating transcriptomic and proteomic data helps scientists understand how these genetic factors translate into disease mechanisms.

“By studying genetic variation across populations, we can determine why diseases affect some more than others and why treatments work differently among individuals,” said Kári Stefánsson, founder and CEO of deCODE genetics. “The combination of genomics, transcriptomics and proteomics allows us to create a more complete picture of human biology and develop medicines specific to each patient.”

For example, Stefánsson’s team collaborated on a genome-wide study of 30,000 patients with autoimmune thyroid disease (AITD) and over 725,000 controls in Iceland and the UK. The disease is common, often inherited, and mainly associated with hypothyroidism, or low thyroid function, when immune cells attack the thyroid gland.

The team discovered a relatively rare variant in the FLT3 (fms-like tyrosine kinase 3) gene that codes for a receptor on immune and blood cells essential for cell growth. They found that people with this DNA variant are at higher risk for autoimmune diseases like AITD, systemic lupus erythematosus (SLE), or lupus, rheumatoid arthritis, celiac disease and nearly double the risk for acute myeloid leukemia (AML).

The team examined participants' transcriptomes, or RNA (the intermediary between a gene and the protein that gene makes), and found that the variant altered the FLT3 gene, resulting in an inactive FLT3 receptor. This is known as a loss of function variant.

Finally, studying the proteome, or proteins, the team found that participants with the variant had increased FLT3 protein levels in their blood. This unexpectedly heightened FLT3 activity, revealing that the loss-of-function variant actually boosted FLT3 receptor function, leading to the development of blood cells involved in AML and autoimmune diseases. Amgen is currently researching the potential of blocking FLT3 signaling in certain autoimmune diseases.

Studies like these improve understanding of disease biology, leading to more conviction in potential targets.

“Freyja,” NVIDIA’s DGX SuperPOD supercomputer is located at Amgen’s research facility in Iceland.

To analyze the massive amounts of omics data available, Amgen uses artificial intelligence (AI) and machine learning (ML) to quickly identify hidden patterns. These insights help prioritize promising drug targets and enhance the design of therapeutics with a greater chance of success.

These kinds of analyses take enormous amounts of computing power. Amgen meets these demands with technologies like NVIDIA's DGX SuperPOD, a state-of-the-art supercomputer in Iceland, named "Freyja" after the Norse goddess of foresight and life.

Learn more about AI at Amgen.

A New Era of Precision Medicine

Integrating omics data into drug development is also reshaping the future of precision medicine. Rather than a one-size-fits-all approach, scientists are now designing treatments that can bring more tailored options to patients.

Take cardiovascular disease, for example. Traditional treatments target broad populations, but many patients still suffer from heart attacks or strokes despite receiving standard care. Using omics, Amgen is developing potential therapies that target specific genetic and protein markers linked to cardiovascular risk—bringing new hope to patients who need more options.

Surfaceomics, a growing field of omics, focuses on discovering new targets on cell surfaces to develop better treatments, particularly for cancer. Amgen scientists are exploring ways to better understand these cell surface molecules, their modifications and their interactions. One promising strategy is dual targeting, where a therapeutic molecule binds to more than one target specific to the surface of tumor cells. This may enhance a therapy’s precision and effectiveness, while reducing harm to healthy cells.

Omics is also helping scientists match the right therapies to the right patients by discovering biomarkers, or molecular signatures, of diseases such as cancers. This ensures that patients receive the most effective treatments, while avoiding unnecessary side effects.

For patients, the impact of omics could be profound. It could mean treatments that are more effective from the start and fewer failed therapies. Instead of settling for trial-and-error, medicine is becoming more predictive and personalized.

Amgen leverages expertise in human biology to drive the next wave of medical innovation. Investing in omics is not just helping to discover new medicines, it’s enhancing the understanding of disease mechanisms and expanding treatment options for patients in need.