Fighting Disease with Biologics
Biologics Research Pushing Frontiers of Science With More Than 900 Medicines and Vaccines in Development
Biologics are developed through biological processes using living cells or organisms.
Biologics are made from a variety of natural sources—human, animal or microorganisms. Like small-molecule drugs, some biologics are intended to treat diseases and medical conditions. Other biologics are used to prevent or diagnose disease. These medicines represent the next exciting frontier of biopharmaceutical research. Millions of people already have benefited
from medicines and vaccines developed through biotechnology. America’s biopharmaceutical research companies are using biological processes to develop 907 medicines and vaccines targeting more than 100 diseases—including 352 for cancer, 188 for infectious diseases, 69 for autoimmune diseases, and 39 for AIDS/HIV and related conditions.
1 in 3,500 Newborn Boys Impacted by DMD
Duchenne Muscular Dystrophy (DMD) is a genetic disorder and the most severe form of muscular dystrophy in childhood. RNA interference therapeutics are an exciting new frontier for the development of novel therapies for patients with genetic disorders. There are several RNAi therapies in clinical trials which have demonstrated potential in treating DMD, including improving DMD patients’ ability to walk.Learn More
These Medicines Often Represent Cutting-edge Research
The latest scientific discoveries are translated into novel therapies that provide new treatment options for patients. Increased understanding of the molecular and genetic bases of disease has opened up the development of a range of targeted treatments. For instance, monoclonal antibodies (mAbs) are proteins that help the immune system identify and bind to foreign substances. Thirty years after initial development, these therapies help treat some of the most costly and challenging diseases.
U.S. pharmaceutical companies account for 80 percent of the world’s R&D in health care biotechnology. America’s biopharmaceutical research ecosystem is uniquely positioned to deliver on the great promise of science if supported by policy and regulatory environments that encourage continued innovation.
The Human Genome
Inside every human body are about 25,000 genes—each responsible for a specific protein. If one of the genes is missing or defective, the body will not have the proteins it needs to function properly and may have proteins that actually cause disease. The modern tools of biotechnology—coupled with computer technology’s ability to analyze massive amounts of data quickly—help biopharmaceutical scientists determine which genes or proteins are defective and are being used to develop new treatments across a range of therapeutic areas.
Americans a Year Die From Lymphoma
But a third-generation antisense medicine in development for the treatment of lymphoma inhibits production of a specific protein which regulates many key genes important in cancer growth. Biologics could be the answer to treating many of these fatal diseases.Learn More
Medicines in the Pipeline
America’s biopharmaceutical research companies are developing 907 biologics targeting more than 100 diseases, including 352 for cancer, 188 for infectious diseases, 69 for autoimmune diseases, and 39 for AIDS/HIV and related conditions.
Monoclonal Antibodies (mAb)
A mAb designed to block the IL-13 cytokine, a protein messenger between cells that triggers inflammation. Blockage of IL-13 may reduce the risk of asthma and other respiratory diseases.
An RNAi-targeted therapeutic in development seeks to restore the function of dystrophin. Early clinical trials of the medicine have demonstrated significantly improved dystrophin expression as well as improvement in DMD patients’ ability to walk.
Stem Cell Therapy
Researchers are exploring transplantation of a patient’s own bone marrow cells into damaged heart tissue to regenerate heart tissue. It is believed that a patient’s immune system will not attack the newly transplanted cells because they are native to the patient.
A gene therapy employs an adeno-associated virus (AAV) as a vector to deliver to the gene neurturin, which has been found to restore cells damaged in Parkinson’s patients and protect them from further degeneration.
View and download additional resources on biologics and biotechnology:
View the full 2013 report on biologics in development.
From Biologic Medicines in Development, 2013
This information is potentially valuable to patients still seeking effective treatments for hundreds of diseases.
Selected Facts about Diseases Targeted by Biotechnology Medicines in Development