Why Gene Therapy Companies need a Quality & Electronic Batch Record System

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Why Gene Therapy Companies need a Quality & Electronic Batch Record System

Gene therapy is on the move. The industry is currently valued at $5.61 Billion, with a $19.2B projection by 2028. It has already advanced the progress of human healthcare impressively. Possibilities include new weapons against many individual inherited disorders, as well as sickle cell anemia and cancer. By the very nature of the domain, information management and control are crucial for safe operation and continual development.

Gene therapy is often based on individual health information, which must be stored, maintained, and made available according to HIPAA and GDPR guidelines.

Gene therapy usually requires investigative and approved products to be done in small batches. Most CDMOs operate on a scale-up basis. The time required to produce a Master Production Record for a small yield gene therapy batch can prove cost-prohibitive. When knowledge transfer, etc., is added to the tab, it often makes sense for the developer to do this in-house.

Gene therapy sponsors also need the power of a full-quality system for their operations.   They must:

  • Establish a quality system for clinical studies and clinical materials
  • Negotiate quality agreements with vendors and contractors
  • Ensure vendors and contractors are qualified
  • Maintain controlled documents
  • Approve specifications for clinical trial materials
  • Review and approve production deviations and out-of-specification investigations
  • Approve clinical trial materials before release to clinic
  • Review and approve QC and stability test results

The FDA has created a draft guidance for both Cell and Gene therapy products because of their complexity. We’ve reviewed the items under consideration and believe they will certainly require a powerful and highly configurable documentation platform.

InstantGMP’s PRO system automates these operations, provides the requisite reconfigurability as the industry requirements evolve, and offers capabilities for compliant PHI maintenance.

For a detailed study of how our system supports an RNA-based cell expert firm, look here.

How can you hear more about this in action? Our next blog will contain an interview with one of our in-house implementation experts on how to put an Electronic Batch Record together for a gene therapy product.

Please reach out to us at www.instantgmp.com to learn more.


“Manufacturing Change and Compatibility for Human Cellular and Gene Therapy Products,” FDA, 7/23

Presentation, “Quality Management Systems for Virtual Companies,” Jeremy Hall, InstantGMP, 11/23