The transformative power of cellular and gene therapy products has ignited new hope in the scientific community for innovative treatments. In the majority of cases, these biologically derived therapies offer hope for otherwise untreatable illnesses, adding to the rising interest in the ever-growing roster of clinical trialsi. Considering how much excitement has developed around these therapies, they are still a relatively new form of medical treatment, as the US approval of the first Advanced Therapy Medicinal Product (ATMP) occurred just eleven years agoii. As human cell and tissue-based products continue to proliferate, international legislative parameters have struggled to keep pace.iii
A unified global standard for each regulatory process step must be enacted to create biologically derived products that are universally safe and efficient.iv In producing biologically derived products, the complexity of culturing human cells makes GMP-compliant manufacturing difficult.v Consequently, the regulatory process from development to clinical trials to FDA approval is a long and arduous journey. In fact, there have only been twenty additional FDA approvals for ATMPs since the first, PROVENGE®, was approved in 2010 for treatment in certain prostate cancers.vi
Although specific laws and regulations will vary by region, an internationally recognized process will require biomanufacturing solutions that support compliance with current GMPs to assure the quality and safety of products for human use.vii
In cell culture
, meeting requirements for cleanrooms
is one of the most critical issues for laboratories adhering to GMP requirements. As regulations become more defined and agreed upon globally, more innovative medicines will reach more patients through proper monitoring and control of GMP manufacturing processes. While the future of international regulations will still evolve for adequate cell and gene therapy manufacturing, signs indicate that a properly maintained cleanroom
will be a global requirement.
Premkumar Jayaraman, Ryan Lim, Jacqueline Ng, Mohan C. Vemuri, "Acceleration of Translational Mesenchymal Stromal Cell Therapy Through Consistent Quality GMP Manufacturing," Front Cell Dev Biol
. 9, no. 648472 (April 2021): 1-19, https://doi:10.3389/fcell.2021.648472
Kazuo Yano, Alessondra T. Speidel, Masayuki Yamato, “Four Food and Drug Administration draft guidance documents and the REGROW Act: A litmus test for future changes in human cell- and tissue-based products regulatory policy in the United States,” Journal of Tissue Engineering and Regenerative Medicine
, 12, no. 7 (July 2018): 1579-1593, https://doi:10.1002/term.2683
Peter G. Childs, Stuart Reid, Manuel Salmeron-Sanchez, Matthew J. Dalby, “Hurdles to uptake of mesenchymal stem cells and their progenitors in therapeutic products,” Biochemical Journal
, J. 477, no. 17 (September 2020): 3349-3366, https://doi:10.1042/BCJ20190382
vJayaraman P, et al., "Acceleration of Translational Mesenchymal Stromal Cell Therapy."
viFDA, "Approved Cellular and Gene Therapy Products.
Ayesha Aijaz, Matthew Li, David Smith, Danika Khong, et al., "Biomanufacturing for clinically advanced cell therapies," Nature Biomedical Engineering
, 2, no. 6 (June 2018): 362-376, https://doi:10.1038/s41551-018-0246-6