Fifty years ago, the idea of altering a gene to treat or even cure a disease was considered science fiction. Today, cell and gene therapy are transforming treatment options for some patients and are part of an exciting new era of medicine.
A new report released today finds there are 289 novel cell and gene therapies in development for a variety of diseases and conditions. The therapies represent the translation of basic scientific insights into innovative new treatment options for patients. These medicines in development are either in clinical trials or awaiting review by the U.S. Food and Drug Administration.
The novel cell and gene therapies in the development pipeline are the result of years of pioneering research. The range of diseases these therapies can address is broad and covers blood and eye disorders, cancer and infectious disease to name a few.
This new field of cell and gene therapy, a major part of the broader field of regenerative medicine, is already making an impact. American Society for Blood and Marrow Transplantation president John F. DiPersio, M.D., Ph.D. noted, “The field of regenerative medicine is enhancing science, education and clinical care that will 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.”
Justin and Boris, a patient and a biopharmaceutical researcher respectively, know firsthand the impact these treatments can have on individual lives. At the age of seven, Justin was diagnosed with acute lymphoblastic leukemia, a type of blood cancer. He spent more than half his life battling this disease, but a new CAR-T cell therapy finally put him in remission and gave him hope.
Boris is particularly interested in advancing the potential of personalized medicine and CAR-T therapy – the same therapy that saved Justin’s life. “It’s being manufactured or generated for every single patient. We teach [white blood cells] how to fight cancer, and give them back to the patient,” he said. While implementation may be complex, researchers like Boris demonstrate the future for cancer patients has never been more promising.
To learn more about the new advancements in cell and gene therapy, read the full report here.
Andrew Powaleny
Andrew Powaleny is the Deputy Vice President of Public Affairs at PhRMA, where he leads communications on scientific, regulatory and intellectual property issues, highlighting the biopharmaceutical industry’s efforts in developing innovative medicines and vaccines while promoting an environment that encourages necessary risk-taking. For five years starting in 2019, he launched and led PhRMA’s grassroots initiative, Voters for Cures, bringing patients and caregivers together to advocate for access to medicine and biopharmaceutical innovation. He also co-founded and co-chaired PRIDE Umbrella, a resource group for LGBTQ+ individuals. Prior to joining PhRMA in 2015, Andrew worked as a communications consultant in Washington, D.C., and as Deputy Press Secretary for the U.S. House Committee on Energy and Commerce under Chairman Fred Upton. He holds a degree in public policy and government from Eastern Connecticut State University, with a minor in music. Outside of work, Andrew enjoys exercise, musical theater and mentoring college students through The Fund for American Studies alumni association.
Andrew Powaleny
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Cell and Gene Therapy
New report shows nearly 300 cell and gene therapies in development
New report shows nearly 300 cell and gene therapies in development