Cellular and gene therapies are one of the hottest product development trends in 2024. They have the potential to revolutionize the healthcare industry, bringing the hope of cures for genetic disorders and rare/ultra rare diseases that have seen few, if any, treatment advances. They also bring the promise of individualized treatment based on the genetic makeup of a patient.
According to the American Society of Gene and Cell Therapy, there are more than 4,000 gene, cell, and RNA therapies being developed around the world. In the US, FDA has approved 38 products and over 1,500 clinical trials are ongoing.
FDA has been building the resources available to sponsors to assist in the development of cell and gene therapies. Recently, FDA reorganized the groups supporting these advanced therapies by creating the Office of Therapeutic Products (OTP) within the Center for Biologics Evaluation and Research (CBER) which includes the Office of Gene Therapy CMC, Office of Cellular Therapy and Human Tissue CMC, Office of Plasma Protein Therapeutics CMC, Office of Clinical Evaluation, Office of Pharmacology/Toxicology, and Office of Review Management and Regulatory Review. The OTP Office structure aligns disciplines by product type and enhances the Agency’s expertise in these highly specialized disciplines.
FDA has provided opportunities to expedite the development of promising cell and gene therapies intended to treat serious conditions through the Regenerative Medicine Advanced Therapy (RMAT) designation. RMAT eligible therapies include cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products. The RMAT program is an additional program that was added to the already existing expedited development programs for serious diseases or conditions including Breakthrough Designation, Priority Review, and Accelerated Approval.
In addition, CBER has released a number of new guidance to support cell and gene therapy development. Since 2020, they have released 16 new draft guidance. The guidance topics are wide ranging covering specific therapeutic areas such as gene therapy for retinal disorders and for hemophilia as well as for broad topics such as CMC Information for Human Gene Therapy INDs. A complete list of recent cell and gene therapy guidance can be found here. These new guidance provide a wealth of information for industry in navigating the quickly evolving regulatory landscape for cell and gene therapy. However, interpretation and implementation of these guidance requires regulatory expertise that can only be accomplished through experience developing these products in real time. Facet has successfully helped a number of cell and gene therapy developers formulate a strategic development plan from concept to clinic as well as secure special regulatory designations for their products. Facet is particularly excited about helping to progress cell and gene therapies because we see enormous potential for the discovery of new therapies for currently untreated debilitating and life-threatening diseases.