The pipeline for new drugs starts here

We find new disease targets and potential new therapies. Our work continues until they’re ready for patient testing.
scientists at work in lab

Translational research

We have the scientific expertise and technology platforms to establish innovative disease targets, identify potential new drugs and advance them toward the clinic.

Our competitive advantages

Outstanding academic research and premier drug discovery science. The Prebys Center is one of the most advanced nonprofit drug discovery centers in the world.

Powerful academic network and collaborative reputation. Partners include universities, clinical organizations, disease foundations, government agencies and biopharma companies.

Success in securing translational research grants. More than $100 million over the past five years.

A track record of successful milestone-driven drug discovery. Sanford Burnham Prebys science has supported many key treatments and tests. See the accompanying facts.

Institute facts

7

FDA-approved treatments and tests

10

current clinical trials underway

35

startups from Sanford Burnham Prebys innovations

60

current projects in the pre-clinical candidate pipeline

Asset 1

Current clinical trials

Supported by Sanford Burnham Prebys research

Immune therapy

Carl Ware, Ph.D.

An investigational immune therapy that came out of collaboration with Eli Lilly and led by Carl Ware, Ph.D., has entered a Phase 1 clinical trial. The compound, LY3361237, inhibits inflammation by activating an immune checkpoint receptor. The results of this trial could have a significant impact on those affected by autoimmune diseases such as lupus, psoriasis and rheumatoid arthritis.

 

Ectopic calcification diseases

José Luis Millán, Ph.D.

José Luis Millán, Ph.D., has led foundational research resulting in a clinical trial for a compound that inhibits calcium deposits in parts of the body where they shouldn’t be, such as blood vessels. The compound, DS-1211, has been licensed to Daiichi Sanko and may benefit those suffering from heart failure, kidney disease and other conditions where vascular calcification creates a health risk.

 

Pancreatic cancer

Erkki Ruoslahti, M.D., Ph.D.

A compound discovered by a research team led by Erkki Ruoslahti, Ph.D., has entered a Phase 1 trial for pancreatic cancer. The compound, CEND-1, is used with existing cancer drugs, helping streamline their deep penetration into solid tumors, which are often surrounded by thick fibrotic tissue. This advance could be also be significant for those suffering from other solid tumor cancers such as brain and ovarian cancer.

 

FDA-approved treatments and tests

Supported by Sanford Burnham Prebys research

To treat anemia caused by chronic renal disease and chemotherapy

Approved in 1989 and 1993

In the US more than 20 million people ages 20 and older have CKD (chronic kidney disease) and roughly 400,000 of these people are undergoing dialysis treatment. Nearly all people on dialysis have anemia. For 25 years over 1.5 million Medicare patients on dialysis who suffer from anemia have been given EPOGEN®, diminishing the need for a blood transfusion. Sanford Burnham Prebys scientists generated the technology that enabled the development of the drug.

Test to screen for prostate cancer

Approved in 1994

The PSA test is the most common screening tool for prostate cancer. The test measures the amount of prostate-specific antigen (PSA) in blood and helps doctors detect prostate cancer, monitor treatment or assess recurrence. Sanford Burnham Prebys invented the technology used in the PSA test.

To prevent blood clots during a heart attack or angioplasty

Approved in 1998

Every 40 seconds, someone in the United States has a heart attack. A heart attack, also called myocardial infarction, occurs when a blood clot completely obstructs a coronary artery supplying blood to the heart muscle. Angioplasty is a procedure that opens blocked arteries and restores normal blood flow to the heart muscle. Sanford Burnham Prebys contributed to the research that enabled the development of Integrilin.

To treat cutaneous T-cell lymphoma

Approved in 1999

Targretin® is used to treat the skin problems arising from cutaneous T-cell lymphoma in patients who have not responded well to other treatments. Cutaneous T-cell lymphomas are a group of disorders characterized by abnormal accumulation of malignant T-cells in the skin potentially causing rashes, plaques and tumors. There are approximately 1,000 new cases of skin lymphoma each year in the U.S. Sanford Burnham Prebys scientists performed research that helped identify the drug.

To prevent blood clots from forming

Approved in 2000

Aggrastat is a type of “blood thinner” prescribed for people at risk of a heart attack or other serious blood flow problems. About 2 million to 3 million people take blood thinners every year to help blood flow smoothly through veins and arteries, and to keep blood clots from forming or getting bigger. Sanford Burnham Prebys scientists performed fundamental research on the biology of blood clotting which led to the development of this drug.

To treat hypophosphatasia or soft bone disease

Approved in 2015

Strensiq® is an innovative enzyme replacement therapy for the treatment of patients with perinatal/infantile and juvenile onset hypophaophatasia (HPP). HPP is a genetic, chronic, progressive and life-threatening metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications. Strensiq® is the first therapy indicated to treat HPP improving bone mineralization and survival rates. 
HPP is a rare disease that occurs in approximately one per 100,000 births. Sanford Burnham Prebys scientist José Luis Millán performed key research on the mechanisms that control normal skeletal and dental mineralization that led to the development of this drug.

To treat chronic lymphocytic leukemia

Approved in 2016

Venclexta™ is used to treat people with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), who have received at least one prior treatment. People treated with Venclexta™ + rituximab were 81% less likely to have their disease worsen, or to die before their disease worsened, compared to another treatment (bendamustine + rituximab (BR). The goal of the treatment is achieving remission. 
The American Cancer Society estimates that more than 20,000 cases of CLL and SLL (combined) occur in the U.S. per year. The condition mainly affects older adults, and the risk is slightly higher in men than women. Sanford Burnham Prebys scientists performed fundamental cancer research that lead to the identification of the drug.