Fact Sheets
SELECTED BIOTECHNOLOGY MEDICINES IN DEVELOPMENT
Asthma – According to the American Academy of Asthma, Allergy and Immunology, 20 million Americans suffer from asthma—one out of four Americans. One human monoclonal antibody in development potentially blocks the activities of two cytokines that play a key role in developing asthma.
Critical Limb Ischemia – Critical limb ischemia (CLI) is a severe blockage in the arteries of the lower extremities that markedly reduces blood flow. CLI is a chronic condition that results in severe pain in the feet or toes, even while resting. Complications of poor circulation can include sores and wounds that won't heal in the legs and feet. Left untreated, the complications of CLI will result in amputation of the affected limb. One medicine in development is a DNA therapy that stimulates the growth of blood vessels and is intended to reduce the need for amputations. A phase II clinical trial in 107 patients has demonstrated a statistically significant reduction in the rate of both major amputations and all amputations in those receiving the medicine compared to those receiving a placebo.
HIV Infection – More than 1 million Americans have been diagnosed with HIV infection since the virus was identified in the 1980s, according to the U.S. Centers for Disease Control and Prevention. According to the National Institute of Allergy and Infectious Diseases, a safe and effective HIV vaccine is critical to the control of HIV globally. There are several DNA vaccines in development that potentially block the proteins and enzymes essential for HIV replication.
Auto-inflammatory Diseases – Traditionally, auto-inflammatory diseases were treated with drugs that suppressed the entire immune system. While newer therapies control disease better, they are often short-acting. One human monoclonal antibody in development has achieved long-lasting remission from inflammatory conditions. Inflammatory symptoms include fever, joint pain and skin rash. The antibody blocks a key chemical messenger that cause inflammation and tissue destruction.
Macular Degeneration – Macular degeneration (AMD) is the leading cause of blindness in the United States for those age 55 and over, and it affects 10 million Americans, according to the American Macular Degeneration Foundation. AMD can either be wet or dry. In wet AMD, new blood vessels grow beneath the retina and leak blood and fluid, causing disruption and dysfunction of the retina and creating blind spots. A new medicine in development for the wet form of AMD is a recombinant fusion protein that binds to the growth factor protein that plays a critical role in blood-vessel formation in the eye. The medicine works by blocking new blood-vessel growth and leakiness.
Muscular Dystrophy – According to the National Institute of Neurologic Disorders and Stroke, muscular dystrophy (MD) is a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. One potential medicine in development for adult MD and other muscle-wasting diseases is a recombinant human antibody (immune system protein). The potential medicine is designed to inhibit the protein myostatin, which limits muscle growth. If approved, it would be the first treatment to halt the breakdown of muscle in MD.
Prostate Cancer – In the United States, prostate cancer is the second leading cause of cancer death in men, and more then 30,000 are expected to die from it this year, according to the American Cancer Society. A potential medicine in development may represent the first in a new class of active cellular immunotherapies (ACIs). ACIs are uniquely designed to stimulate a patient’s own immune system to fight cancer. The medicine under study targets the prostate cancer antigen, prostatic acid phosphatase, which is found in nearly 95 percent of prostate cancers.
Rheumatoid Arthritis – Rheumatoid arthritis affects 2.1 million Americans, most of them women, according the Arthritis Foundation. In this autoimmune disorder, the joints become painful, swollen, stiff and, in severe cases, deformed. One medicine in development is a recombinant version of human alpha-fetoprotein (hAFP), an immunomodulatory serum protein that, at high levels, has long been associated with remission of many autoimmune diseases. In clinical trials, the medicine has been well tolerated and supports the idea that hAFP may be an important new therapeutic option in the treatment of rheumatoid arthritis and other autoimmune disorders. The medicine is also being studied in additional autoimmune disorders.
Spinal Cord Injury – An estimated 11,000 new cases of spinal cord injury occur each year. The number of people living today with a spinal cord injury ranges from 225,000 to 288,000, according to the Spinal Cord Injury Information Network. One monoclonal antibody is in development for potential use in the regeneration of corticospinal tract fibers resulting from an acute spinal injury. The antibody neutralizes a protein that inhibits growth of spinal fibers.

