Cell and gene therapy (CGT) innovators are focusing on increasingly complex diseases and indications. The preference for off-the-shelf allogeneic products is still present, but developers also are not shying away from providing autologous products exclusively. Although most CGT companies have established processes, they still depend heavily on manual operations and single-source supply chains. Thus, companies continue to seek out technologies that can make cell therapy processing better and faster. As the number of cell therapy clinical trials increases, such technologies will need to be automated and scalable. Equally important, manufacturers will need better control of their processes and better assurance of their supply chains. Articles in this featured report identify key areas of need for cell therapy processing. Read the articles below to learn more about the obstacles that cell therapy manufacturers are facing as a result of rapid growth, the COVID-19 pandemic, and lack of standardization.

Introduction: Preparing for Process Improvement — Discussions of the Cell Therapy Industry’s Supply Chain, Automation, and Control Needs
by Maribel Rios and Brian Gazaille, with Miguel Forte, Fabian Gerlinghaus, and Kwok Pang
BPI’s managing and associate editors kick off the featured report with interviews that call attention to business and technical hurdles that must be negotiated for the cell therapy industry to realize its full potential. First, experts from two 2020 industry conferences explore emerging challenges with supply chains — particularly for starting and raw materials — in the wake of rapid industry growth and the COVID-19 pandemic. Then, the editors speak with representatives from two CGT industry suppliers to learn how concurrent advances in automation technologies and closed cell therapy processing are increasing process scalability and improving batch traceability.

Untapped Potential of Tissue Engineering: The Three Obstacles Holding It Back
by Lev Gerlovin, Andrew Thomson, and Jack Vailas
Tissue-engineered medicinal products (TEMPs) lag behind CGTs in terms of investment and number of marketed products. Part of the reason for the holdup is that tissue therapies are much more complex in structural composition and processing requirements. Herein, authors from Charles River Associates discuss three main areas that present difficulties for current tissue therapy developers: technical complexity, a lack of clear regulatory pathways for TEMPs, and insufficient reimbursement models.

The Difficulties of Manufacturing Cell and Gene Therapies At Scale
by Allan Bream and Brita Salzmann
Despite the excitement surrounding advanced therapies, developers and manufacturers of such products remain anxious about several challenges relating to CGT facilities, production capacity, and operations. Here, authors from CRB Group share and analyze data from their survey of nearly 150 CGT industry leaders. Their findings show a clear preference among manufacturers of advanced therapies for design and construction of flexible facilities that can adapt rapidly and frequently to future process improvements and technologies. That transition to flexible manufacturing could enable biomanufacturers to prepare for scale-up or scale-out to GMP commercial manufacturing as demand for novel therapies continues to grow.

Manufacture and Regulation of Cell, Gene, and Tissue Therapies: Part 2 — Regulatory Guidance
by Anjali Apte, Adeyemi Afuwape, Zalinka Buljovic, and Zeb Younes
Regardless of region, regulatory agencies expect developers of advanced therapies to comply with current legislation on good manufacturing practices (GMPs), good clinical practices (GCPs), and good laboratory practices (GLPs), with some flexibility permitted according to the developmental stage of their products. But because more ATMPs are targeted toward diseases with high unmet medical needs, and because small- and medium-sized companies and academia typically pioneer those products, regulatory agencies also provide expedited pathways and incentives to bring such medicines to patients. Read this article, which continues discussion from BPI’s November–December 2020 Featured Report, to learn about different ways that American, European, and Japanese regulatory agencies support developers of advanced therapies, especially regarding orphan drugs and other exceptional cases.