PhRMA Highlights Impact of Cell and Gene Therapies in New Report

scientist using tweezers on animated strand of DNA

Over the past few years, cell and gene therapies have become significant components in the R&D spending of numerous drug companies. And more funding is likely to be devoted to that segment as more and more therapies are approved by global regulatory agencies.

This morning the Pharmaceutical Research and Manufacturers of America (PhRMA) released a report that shows there are nearly 300 novel cell and gene therapies currently in development throughout the industry. The therapies are being studied as treatments for a variety of diseases and conditions. The range of diseases these therapies can address is broad and covers blood and eye disorders, cancer and infectious disease to name a few, according to the report.

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Cancer is by far the leading recipient of cellular and gene therapy research. According to the report, of the 289 therapies currently in development, there are 111 different types aimed at cancer. The report does not break down the types of cancer that are the subject of these therapies. Perhaps the most widely known therapy of this type for cancer is CAR-T. So far the U.S. Food and Drug Administration has approved two CAR-T treatments for blood cancers. Multiple companies are working on similar treatments for other cancers, as well as SPEAR-T treatments for solid tumors. The first CAR-T cancer therapies were approved by the FDA last year.

In the PhRMA report, the story of Justin is shared. At the age of 7, Justin was diagnosed with acute lymphoblastic leukemia, a blood cancer. Justin was treated with a CAR-T treatment and his cancer is now in remission.

“Justin remains resilient and hopeful for his own future, as the treatment has given him new hope, even with the setbacks he’s faced. Now, other patients and families can have hope for their path to longer, healthier lives as well,” the PhRMA report said.

The next leading therapeutic target for these therapies are in the eye. There are 28 different cell and gene therapies in pipelines right now for eye disorders. Almost one year ago, the FDA approved Spark Therapeutics' Luxturna, a gene therapy for a rare, genetic form of blindness. Luxturna was the first FDA-approved gene therapy that targets a disease caused by mutations in a specific gene.

Blood disorders, cardiovascular disease and neurologic disorders are also a common focus of cell and gene therapies. The report highlights several treatments that are in development, including Novartis’ gene therapy treatment for spinal muscular atrophy, which was granted priority review by the FD last week.

The therapies target DNA or RNA. As the PhRMA report notes, both approaches “seek to modify genetic material to improve functioning or fight disease.” Gene therapies use genetic materials to manipulate the cells of a patient. There are multiple types of gene therapies in development. These therapies seek to replace a mutated gene that is the cause of a genetic disease.

Cell therapy is slightly different. That procedure involves the infusion or transplanting of whole new cells, such as stem cells or dendritic cells, into a patient who is battling a disease. Those new cells are used to help replace or repair damaged cells in order to help the body fight a disease.

In the report, PhRMA noted that the field of gene and cell therapy is making an impact in regenerative medicine. John F. DiPersio, president of the American Society for Blood and Marrow Transplantation, said regenerative medicine is “enhancing science, education and clinical care” in order to “provide patients with expanded and genetically modified stem and immune effector cells for the treatment of inherited diseases, solid tumors and hematologic malignancies to live longer lives.”

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