Cell and gene therapies for treating multiple myeloma, such as CAR-T, have resulted in breakthrough health outcomes for patients. In the last decade, approval of cell and gene therapy (CGT) products has increased significantly. Multiple other products are in clinical development for solid tumors, genetic deficiencies, and infections that promise more effective treatments to patients.
Applying the required Chemistry, Manufacturing and Controls (CMC) is critical to expediting product approval. This blog post will dive into the essential CMC requirements for cell and gene therapy products.
Cell and Gene Therapy
Cell therapy involves implanting human cells into the body to replace or repair damaged ones. This is done to perform a specific function, such as treating diseases or conditions. Gene therapy changes genes inside a patient’s cells to treat, prevent or cure a disease.
Cell and Gene Therapy CMC Requirements
Modifying human cells and genes is a complex process in which many components must be considered and controlled. The FDA released an updated guidance document in 2020 entitled “Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Guidance for Industry.” Their guidance is designed to help sponsors of gene therapy INDs provide the correct CMC information and ensure their product and drug development methods are safe1.
The main areas that must be addressed during INDs include characterizing manufacturing components and materials, product manufacturing procedures and product testing. These areas must meet standards to ensure the manufacturing process consistently results in products that are safe and efficacious to patients.
Characterization of Manufacturing Materials
The materials used to develop CGT products must be of a high standard to meet regulatory requirements. Some of the unique manufacturing components that may need to be considered are:
- Plasmids
- Viral Vectors
- Host cells
- Reagents
Product Manufacturing Processes
Thorough descriptions of processes must be provided as well as extensive details on process controls and testing methods. Examples unique to CGT products include:
- Vector Production / Purification
- Chain-of-Custody / Chain-of-Identity
- Storage and transportation
- Final formulation
Product Testing
In-process and final product release testing needs to be identified and specifications established. Standard tests would include:
- Cell number or dose
- Identity
- Microbial testing
- Potency
- Purity
- Viability
The FDA requires these standards to be met to ensure patient safety. DSI can provide further information on the FDA document and the areas mentioned above.
DS InPharmatics Cell and Gene Therapy Services
DSI consults provide a range of services to suit your company’s needs, ranging from regulatory affairs to CMC and quality assurance. Please don’t hesitate to contact us to discuss requirements and how our team can support your success.
