July 24, 2018

What if a patient could use her own cells to treat her disease? Bench-to-bedside therapeutics are possible with cell and gene therapies, and there has been steady growth in these industries over the past few years. According to the Alliance of Regenerative Medicine, there are over 871 regenerative medicine companies worldwide, compared to 672 in 2015.[1] Clinical advancement of these innovative technologies is also experiencing steady growth: 631 underway in 2015, compared to 900+ reported ongoing studies by the end of 2017.[2]

The advancement of cell and gene therapies can be influenced by regulatory authorities, academic leaders, and the regenerative medicine industry. The advancement of regenerative medicine companies rely on collaborations between academia and industry to provide the expertise and resources to bring these technologies to the patient. While over half of clinical trials were sponsored by academic institutions, all gene and cell therapies that received market approval were sponsored by an industry sponsor.[3] These relationships will be key to translating cell and gene therapies from academic settings into the clinic, as the former may not have experience with regulatory procedures.[4]

Early investigations of clinical trials in regenerative medicine were relatively slow. For example, a study on European clinical trials identified 278 gene and cell studies from 2004-2014.[3] During this time, peaks in the number of trials were observed in 2007 and 2009, which may be correlated with regulations for the industry: the EU directive 2001/83/EC that defines the gene and cell products was renewed in 2007 to adopt tissue engineering products, and regulation for gene and cell based therapies EC 1394/2007 was enforced at the end of 2008. Regulation agencies across the globe also differ between jurisdictions for approval.[4] Over the last decade, specific laws for the approval of cell and gene therapies were enacted in Japan and the EU, while in Korea, the US, and Canada these technologies are still regulated under those for biologics. Gene therapies and those used as combinational products are categorized under medicinal products and need to obtain approval in all jurisdictions, while cell therapies require approval under specific subclasses depending on the scope, including:

  • The extent of manipulation
  • Use of homologous cells
  • Indication of local or systemic effect and the known mechanism of action
  • Development of cell therapies by an academic center or industry

Cell and gene therapies face unique challenges, including expensive preclinical and clinical studies, navigating new regulatory pathways, dosing considerations, manufacturing, storage, and shelf-life. The FDA recently released a guidance for cell and gene therapy manufacturing, as product lots may be relatively small and highly variable.[5] Additional manufacturing and quality regulations are implemented for products using human cells or tissue, however no universal global standard for GCP and GMP regulations have been implemented to date. Unfortunately, regenerative medicine clinical studies cannot currently rely on a standard benchmark for trial design or cell therapy development. Randomized controlled clinical trial design is preferred for most drug therapeutics, but regenerative medicine trials often must consider invasive delivery methods, small patient populations, and low alternative treatment pools. In addition, the transfer of preclinical data to clinical protocols may not useful as it is not yet feasible to conduct traditional preclinical pharmacokinetic studies with cell and gene therapies. Instead, clinical protocols for these therapeutics may incorporate aspects to help foster further product development, such as incorporating features of Phase II design into Phase I studies gather preliminary evidence of effectiveness.[6]

At Neox, we will use our relationships with regulatory authorities and reputable manufacturing facilities to identify efficient clinical strategies for regenerative medicine therapies. Years of clinical research experience, understanding local requirements and established relationships with investigators illustrates our ability to maneuver the regulations and protocol design within this exciting field. As a full service CRO, we hold over 15 years of experience working with clients ranging from biotech startups and big pharma, providing tailorable solutions for our clients. Clinical trials continue to move towards globalization. The resources that we have been building in the past 15 years allow us to work all across Europe to include studies in Poland, Germany, UK, the Netherlands, and other countries. Please reach out if you are interested in learning more.



Mary Nguyen, Ph.D.
Business Developer & Project Manager for Business Expansion
[email protected]

4660 La Jolla Village Drive
San Diego, CA 92122, United States

Cited references:
[3] de Wilde, S., Guchelaar, H.-J., Zandvliet, M. L. & Meij, P. Clinical development of gene- and cell-based therapies: overview of the European landscape. Mol. Ther. – Methods Clin. Dev. 3, 16073 (2016).
[4] G.M., C. D., L., B. M., G.M., L. H. & Jarno, H. Global Regulatory Differences for Gene‐ and Cell‐Based Therapies: Consequences and Implications for Patient Access and Therapeutic Innovation. Clin. Pharmacol. Ther. 103, 120–127 (2017).



About the Author and BIOCOM CRO Board Member