Cell Therapies

Measure Twice, Treat Once: Navigating the Regulatory Landscape of Assay Development to Ensure High-Quality CGT Products

Cell and gene therapies (CGTs) are a novel and fast-growing class of transformative therapies designed to address gaps in traditional treatment strategies of some of the most severe diseases. By definition, gene therapy “seeks to modify or manipulate expression of a gene to alter the biological properties of living cells for therapeutic use” (1). That can be either an in vivo delivery of a gene or delivery of a gene to a patient’s cells that are manipulated outside of the…

AAV Vector Manufacturing Platform Selection and Product Development

Adenoassociated virus (AAV) vectors have emerged as the prominent delivery mechanisms of corrective gene therapies. Three such products — Glybera (alipogene tiparvovec, uniQure), Luxturna (voretigene neparvovec-rzyl, Spark Therapeutics), and Zolgensma (onasemnogene abeparvovec-xioi, AveXis) — have been licensed, and a growing number of candidates are entering late-stage development. In mapping out an AAV gene therapy product development strategy, biomanufacturers should address fundamental considerations for their manufacturing strategies for both phase 1–2 clinical evaluation and translation for commercial market supply. A manufacturing…

The Cell Therapy Industry Needs High-Quality Healthy-Donor Material

The cell and gene therapy (CGT) segment has grown tremendously over the past decade. And while the industry deals with a steep learning curve inherent to a rapidly developing field, problems must be solved, and solutions must be reduced to practice. One such problem, long understood by the industry but now thrust into the spotlight, is sample handling: proper collection, processing, preservation, storage, and transportation of cellular starting material. Suboptimal techniques for such logistics have been shown not only to…

Bioprinting Capabilities and Futures

Bioprinting has advanced rapidly through engineering step changes in the use of three-dimensional (3D) printing. With these developments, living cells can be positioned layer by layer to produce functional tissue structures. Key attributes of this emerging technology are its high scalability and modularity, which enable automated and repeatable manufacture of a wide variety of tissues. These high-throughput biofabrication capabilities equip companies with tools to develop 3D-printed tissues for broad applications, from in vitro drug testing models to therapeutic tissue implants,…

Forward Thinking — Evolving the Cell and Gene Therapy Industry: Modern CAR T-Cell Process Addresses Future Demand

It is an exciting time to be involved in the cell and gene therapy industry. We have come to a tipping point where we have shown that the science works. Now we need to industrialize and standardize it, so we can get these life-saving therapies to more patients. As an industry, we can learn from all the experience in the biologics space. With monoclonal antibodies (MAbs), we have transitioned from a manual process to a more standardized and automated set-up.…

Production of Transient Lentiviral Vectors in HEK 293T Cells: Cultivation on Fibra-Cel Disks in a Single-Use, Packed-Bed, Stirred-Tank Bioreactor

Although demand for lentiviral vectors (LVs) for cell and gene therapy is increasing, the standard two-dimensional culture systems used to produce LVs present significant disadvantages. Current bottlenecks in LV production are caused mainly by such disadvantages. Switching to use of bioreactors can eliminate those problems because bioreactors offer the benefits of process automation, tight regulation of production conditions, and reduced labor input. The study reported herein was carried out by the group of David Parsons at the University of Adelaide.…

Streamlining the Path to Clinical Manufacturing

Careful consideration of lot size is crucial for multiyear success of a cell therapy business. RoosterBio’s product design and acceleration business works with the company’s customers to help make plans that are appropriate for their stages of product and clinical development. We use the following major considerations in creating a sound multiyear strategy with intermediate milestones: Understand what the future looks like and work backward Use reasonable to conservative assumptions to estimate a range of needs Invest in the right…

eBook: Autologous Cell Therapies: Commercialization Strategies

Autologous cell therapies are derived from a patient’s own stem cells, typically collected from bone marrow. Those cells are then cultured, expanded, and reinfused back into the patient. Unlike allogeneic cell therapies, this process is repeated for each dose and for one patient. The one-to-one process carries several challenges to commercialization, including high development costs, the need to control the risks of manual processing, and compliance with strict timelines. This eBook presents two perspectives on addressing these challenges. The first…

Toward Standardized Sample Collections: UK Government Seeks to Expand Cell and Gene Therapy Use

The SAMPLE program — a Standard Approach to ATMP Tissue collection — is intended to help build capacity for the UK National Health System (NHS) to expand the use of next-generation cell and gene therapies for both cancer and noncancer illnesses. Now it has been given the green light by Innovate UK (IUK), the United Kingdom’s technology strategy agency. An award through the Industrial Strategy Challenge Fund is supporting the program for standardizing how cell and gene samples are collected…

Immunotherapy: Taking Aim at Solid Cancers

As cell and gene therapies arrive on the market, all eyes have focused on autologous chimeric antigen receptor (CAR) T – cell therapies. At the 2019 Phacilitate Leaders World and Stem Cell Summit in Miami, FL, delegates looked at where the biopharmaceutical industry is going in the cellular immunotherapy space. Whether for off-the-shelf CAR T-cell products, personalized cancer vaccines, or modified natural killer (NK) cells derived from human induced pluripotent stem cells (iPSCs) — cell and gene therapy development is…