In 2017, the U.S. approved its first cell and gene therapy, making the idea of altering a gene to cure or treat a disease a reality. As the increasing pace of R&D propels cell and gene therapy forward, patients can expect more personalized treatments on the horizon. A new report released today finds that there are 362 investigational cell and gene therapies currently in clinical development – a 20% increase since 2018.
Among the cell and gene therapies in development are potential treatments for:
- A gene therapy using adeno-associated virus (AAV)-factor VIII is designed to stimulate the production of factor VIII for the treatment of hemophilia A.
- A gene therapy using AAV vectors is delivering a high-activity Factor IX gene to the liver for the treatment of hemophilia B.
- A second-generation CAR-T cell therapy comprised of genetically-modified T-cells, is designed to target B-cell maturation antigen and to redirect the T-cells to recognize and kill malignant myeloma cells.
- A gene therapy for the treatment of Stargardt disease delivers a corrected version of the ABCR gene directly in the photoreceptors in the retina.
- A gene therapy uses a recombinant AAV9 capsid to deliver a shortened version of human dystrophin to treat Duchenne muscular dystrophy.
While there are hundreds of potential cell and gene therapies in the pipeline, a few of these innovative medicines have already been approved by the U.S. Food and Drug Administration (FDA) and are helping patients today. Currently, there are six diseases currently treated with gene or cell therapy for the treatment of cancer, eye diseases and rare hereditary diseases.
These cell and gene therapies are revolutionizing medicine, but scientists and manufacturers still face technical and logistical challenges to bring these treatments to market in sufficient commercial quantities. A new Oxford Economics paper explores these challenges and ultimately suggests that large capital investments are likely needed to bring future breakthroughs that will standardize processes, simplify delivery logistics, boost efficiency and increase competition. Fortunately, America’s biopharmaceutical companies stand committed to overcoming these challenges in the pursuit of needed breakthroughs for patients.
As researchers explore next-generation therapies, we need to bring similar innovation to payment methods to help ensure patients have access to cell and gene therapies. To that end, biopharmaceutical companies are collaborating with insurers to develop innovative contracting arrangements. These can include results-based or value-based contracts which focus on real-world results for patients and can improve outcomes and alternative financing arrangements to help limit or spread costs over time for a payor. However, outdated federal rules and policies can create uncertainty for manufacturers and may limit the growth and expansion of innovative contracting arrangements.
Novel cell and gene therapies in the development pipeline today are the result of pioneering research by America’s biopharmaceutical research companies. It is crucial we foster an environment that encourages continued innovation in this space. To learn more about cell and gene therapies in development, click here.
Andrew Powaleny is Senior Director of Public Affairs at PhRMA and leads the organization's scientific communications. Before joining PhRMA in 2015, he worked in public affairs for a small firm in Washington, DC and served as Deputy Press Secretary for the House Committee on Energy and Commerce. Andrew came to Washington, D.C. via Connecticut with a degree from Eastern Connecticut State University where he majored in public policy and government. Andrew is active as a runner and volunteer with the DC Front Runners; most recently serving on its Board of Directors for three years as co-race director. He is also a member of the NLGJA: The Association of LGBTQ Journalists and mentors students through his alumni association with The Fund for American Studies. Andrew is passionate about scientific innovation, especially for mental illness, and his heroes are the men and women of America’s biopharmaceutical research companies.