On January 28, 2020, the United States Food and Drug Administration (FDA) announced the release of six final guidances on gene therapy manufacturing and the clinical development of new products. In addition, they published a draft guidance on the interpretation of gene therapy products’ sameness under the orphan drug regulations. With these new guidance documents, the FDA hopes to encourage innovation and advance the product development of gene therapies. The release of these new guidance documents is excellent news for contract research organizations like QPS, which excels in advancing cell and gene therapy research with precision technology and custom-built quantitation methods.
Understanding Gene Therapy
A gene is a unit of DNA that contains hereditary information and instructions for making protein molecules, which play a critical role in the function and maintenance of the human body. During gene therapy, researchers introduce, replace, or inactivate genetic material into cells to treat, modify, or cure a disease.
As of January 2020, the FDA has approved just four gene therapy products, but many more are anticipated to be approved in the coming years. In fact, there are currently more than 900 investigational new drug (IND) applications for ongoing clinical studies in the field of gene therapy. This surge in gene therapy research and development is exciting to physicians, scientists, and regulators, as well as patients. Gene therapy provides hope for many patients suffering from diseases with unmet medical needs, including autoimmune diseases and rare genetic disorders.
The FDA’s Final Guidances and Draft Guidance on Gene Therapy
With the six final guidances published in late January, the FDA hopes to support innovators developing new medical products for people around the world. Providing regulatory clarity for product developers, the guidances offer recommendations to ensure that new products meet the FDA’s standards for safety and effectiveness.
The new draft guidance focuses on how to evaluate differences between gene therapy products that are developed to treat the same disease. With the draft guidance, the agency hopes to give product developers insight into their reasoning regarding the sameness of gene therapy products. In addition, the FDA wants to encourage the development of multiple gene therapy products to treat the same disease or medical condition, which could benefit patients by creating a more competitive market with more choices available across therapy areas.
If your development team is working on a gene therapy solution, QPS may be able to help you advance your product’s development in this emerging area of medicine. We provide gene therapy research using state-of-the-art technology and custom quantification methods, and our scientists will work with your team to determine specific study parameters and design. Contact us today to get started.
Since 1995, QPS has provided discovery, preclinical, and clinical drug development services. An award-winning leader focused on bioanalytics and clinical trials, QPS is known for proven quality standards, technical expertise, a flexible approach to research, client satisfaction and turnkey laboratories and facilities. For more information, visit www.qps.com or email [email protected].