When it comes to GMP considerations, what are the regulatory requirements for cell and gene therapies?

Cell therapies consist of autologous and allogeneic products. Autologous products are taken from an individual patient, modified, and then returned back to the patient in an effort to treat disease while allogeneic products are composed of donor cells delivered to a small patient population, or even just a single patient.

Gene therapies involve modifying a patient’s genes to treat a condition. 

Cell and gene therapy products present manufacturing challenges due to smaller batch sizes, short production turnaround, and the varying nature of the material (human cells and genes) itself. Supply chain challenges include limited raw material suppliers, multiple vendors producing critical components, ensuring the cold chain, and shipping validation.

FDA Cell and Gene Therapy GMP Regulations

As far as U.S. regulations, these therapies fall under U.S. 21 CFR 210 and 211 CGMP regulations and also additional biologic regulations under 21 CFR 600-680 and 1271 that address human cells, tissues, and tissue-based products.

Cell and gene therapy manufacturers can face three different types of FDA inspections:

  1. Prelicensing inspection for a new product during the last part of the Biologics License Application (BLA) review
  2. Preapproval inspection if a change has occurred following approval
  3. Biannual surveillance inspection performed by Team Biologics within the FDA’s Office of Regulatory Affairs

Generally, FDA inspections of cell and gene therapy manufacturers look at the following:

  • Quality
  • Production
  • Facilities and equipment
  • Materials
  • Laboratory controls
  • Packaging and labeling
  • Donor eligibility (primarily for allogeneic products)

Additionally, in recent years, FDA reviewers have expressed concerns about potential data integrity issues involving gene therapy submissions.

Cell and gene therapy manufacturers would be well advised to ensure these seven areas adhere to CGMP practices and requirements.

With Redica, you can filter enforcement action search results by “Cell and Gene Therapies.” Cell and gene therapy manufacturers can use our advanced analytics for better inspection preparation and vendor monitoring. Contact us today to learn more!

Cell and Gene Enforcement Actions
Redica Systems Enforcement Analytics | Cell and Gene Therapy Enforcement Actions

Additional Cell and Gene Therapy GMP Resources

FDA Addresses Cell and Gene Therapy Manufacturing Challenges

Overcoming CMC Complexity for Cell & Gene Products

FDA Takes Quality Systems Approach to CGT Inspections

Data Integrity Concerns Discovered in Gene Therapy Product Submissions

Webinar – CMC Considerations for Cell and Gene Therapy Products

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