Regulatory Update: Cell and Gene Therapy

Commercializing Gene and Cellular Therapy

BY Kimberley Buytaert-Hoefen, Principal Consultant, Parexel - 11.25.19 -

Gene and cellular therapy manufacturing requires strict control in current Good Manufacturing Practice (cGMP) facilities.  Designing a facility or finding a contract manufacturer to meet the manufacturing needs is essential for the success of gene and cellular therapy products. 

Gene and cellular therapy manufacturing facilities include the manufacturing space, the storage warehouse for raw and finished products and laboratory areas.  The manufacturing facilities must be designed for aseptic processing.  Development of fully enclosed manufacturing equipment and built-in controls for in-process testing is optimal for this type of processing. 

Process flow needs to be considered from clinical to commercial manufacturing using a Quality by Design model to validate the cGMP manufacturing process.  This involves defining the critical process parameters, process parameters and in-process tests.  This information should originate from development studies and be used to define a Design Space for the validation of the process.  These parameters and tests should be continuously monitored as part of an on-going process validation.  This can lead to regulatory flexibility to operate in the defined Design Space and to reduced testing as a result of quality being built into the design of the manufacturing process.

A disposable manufacturing model is ideal for gene and cellular therapy manufacturing processes.  This reduces cleaning verification and validation activities.  Cleaning validations can be optimized by using a matrix model for products and equipment.  Other important validation and verification activities include: qualifying the manufacturing facilities and equipment, developing facility cleaning validations, analytical testing and method validations, environmental monitoring programs, batch records development and an on-going process validation plan.  These validation and verification cGMP requirements should be met with the incorporation of Data Integrity best practices for manufacturing and the laboratory using Attributable, Legible, Contemporaneous, Original, Accurate (ALCOA) documentation, aseptic processing and chain of custody best practices.

Incorporating cGMP into the manufacturing environment is required by regulatory authorities to ensure safety and efficacy of gene and cellular therapy products.  The pathway from early concept discovery through to pre-clinical, clinical and commercial manufacturing including the application process for Initial Targeted Engagement for Regulatory Advice (INTERACT) for Center for Biologics Evaluation and Research (CBER) products, Investigational New Drug (IND) and Biological License Application (BLA) are all important components for gene and cellular therapy products which are often approved for Fast Track status.

Parexel Gene and Cellular Therapy experts can guide you through this expeditious process from early concept discovery through to commercial manufacturing in order to bring your lifesaving gene and cellular therapy products to patients in need. 

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