Raw and Starting Materials – ForewordPublished: April 1, 2019
Raw and Starting Materials
Robert G Piperno
Robert G Piperno is the Director of CGT Quality Assurance in the Global Pharma QA organization. In this role he is responsible for providing the global quality oversight and management of internal and external activities supporting clinical and commercial Cell and Gene Therapy Products, including the approval of STRIMVELIS™ by EMA. Rob joined GSK in 2009 as part of the Corporate Audit and Assurance function and transitioned to R&D QA in 2012, and to CGT QA in 2014. He has over 20 years of experience in the pharmaceutical industry and prior to GSK he was employed at Johnson & Johnson for 9 years holding positions including, site quality operations and leading a corporate team supporting the management of external partners and CMOs. Rob has a Bachelor’s and Master’s degree in Biology from Rutgers University and an MBA in Pharmaceutical Management from St. Joseph’s University.DOI: 10.18609/cgti.2019.015
Citation: Cell & Gene Therapy Insights 2019; 5(2), 147-148.
The progression of cell and gene therapy (CGT) products from academic to fully commercial drug products has presented challenges in scalability, production capacity and cost of goods. The growth has also presented new challenges, in this new product space, for starting and raw materials as highlighted in the articles within this edition of Cell & Gene Therapy Insights.
Starting materials include plasmids and cells from donors for autologous and allogeneic CGT products. Both present challenges that the pharmaceutical companies face that are different from the historical ways of working for small molecule and biopharmaceutical products. Plasmids come in both High Quality (HQ) and GMP grade – the availability of the different grades of plasmids requires that pharmaceutical companies develop a strategy for the use of HQ plasmids and a transition to GMP grade plasmids as the drug product progresses to late-stage clinical and commercial supply.
Donor cells present a unique set of challenges to CGT processes as they are inherently variable due to donor-to-donor differences. Process development and validation need to take into consideration the variability to ensure a consistent drug product. Also, the setting of in-process and release specifications on the drug product need careful consideration to ensure the quality of the product while providing for the variability of the donor cells.
The use of healthy donor cells in analytical and process development can cause slight variations from the patient cells used to make the drug product. These potential variations are not always known during the development work and may only become apparent in the drug product manufacturing and testing processes.
Raw Materials for CGT products have similar challenges as other product types, including traceability, BSE/TSE, and the use of human source, with the additional challenge of more of them being less well defined or from single source vendors. Additionally, consideration should be given early in the development process to ensure raw material supplies through development and into commercialization.
FINANCIAL & COMPETING INTERESTS DISCLOSURE
The author has no relevant financial involvement with an organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock options or ownership, expert testimony, grants or patents received or pending, or royalties. No writing assistance was utilized in the production of this manuscript.
Robert G Piperno
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