October 2016 Supplement

October Supplement From the Editor

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Good things come in small packages — like this issue, for example. Each author brings a critical aspect of regenerative medicine development into perspective, highlighting progress and remaining challenges. I am especially pleased to highlight the current stage of gene therapies. At cell therapy conferences a few years ago, some people insisted to me that quality by design (QbD) would have little to no relevance for these advanced therapies. Speakers were academic and medical researchers or commercial manufacturers of traditional…

October 17, 2016

Progress Toward Commercial Scale and Efficiency in Cell Therapy Bioprocessing

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Regenerative medicine includes both cell and gene therapies. Currently 672 regenerative medicine companies operate around the world, and 20 products have been approved by the US Food and Drug Administration (FDA). Of 631 ongoing clinical trials by the end of 2015 (1), over 40% are in oncology, followed in prominence by cardiovascular and infectious diseases. Here I focus on gene and cell therapy bioprocessing in which the final products delivered to patients are cells. Cell therapies are either autologous (derived…

October 17, 2016

Manufacturing Plasmid DNA: Ensuring Adequate Supplies for Gene and Cell Therapies

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The concept of gene therapy is far from new, with initial studies performed over 20 years ago (1). However, in the past few years an explosion of interest in this area has gone beyond initial regenerative approaches using viral vectors. Interest is now moving increasingly into potential use of T cells modified using recombinant viral vectors for immunotherapy applications. Such therapies are based on using either adenoassociated virus (AAV) or lentivirus (1), both vectors being frequently generated through transient expression…

October 17, 2016

Innovative Downstream Purification Solutions for Viral Vectors: Enabling Platform Approaches to Advance Gene Therapies

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Over the past decade, gene therapy applications and their importance in the biopharmaceutical industry have been increasing. Gene therapies promise versatile treatment options that could revolutionize and transform medicine. As treatment modalities, they offer the possibility of long-term and potentially curative benefits to patients with genetic or acquired diseases. Gene therapies are designed to treat disease by delivering genetic material that encodes a protein with a therapeutic effect into a patient’s cells. It can be used to replace a missing…

October 17, 2016

Designing the Optimal Manufacturing Strategy for an Adherent Allogeneic Cell Therapy

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Cell therapies (CTs) offer potential treatments for a wide range of medical conditions (1–6) by replacing cells, repairing tissues affected by either disease or damage (7), or delivering genetic or molecular agents that promote self-healing (8). CT research and development is continuously growing (9), with increasing numbers of CT candidates reaching phase 3 clinical trials (9–11). Developers aim to make products that can survive in a competitive landscape while complying with stringent regulatory requirements to control the quality and safety…

October 17, 2016