Personalized Medicine

Clinical Supply Chain: A Four-Dimensional Mission

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A clinical supply chain fulfills perfectly all four characteristics of what Packowski describes as a “VUCA†(volatility, uncertainty, complexity, and ambiguity) world (1). In commercial markets, supply chains depend predominantly on consumer orders. For global drug development programs, both investigators and patients can be considered end consumers. The international journey of a specific investigational medicinal product (IMP) includes all of the following: global sourcing of comparators, manufacturing, storage, distribution, site/patient (consumer) management, and return and destruction of the IMP. Application…

March 11, 2016

The Potential Application of Real‑Time Release Testing for the Biomanufacture of Autologous Cell‑Based Immunotherapies

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Cell-based immunotherapies (iTx) are emerging as a truly transformative therapeutic modality that is both complementary and convergent with existing regenerative medicine approaches, including gene therapy, cell therapy, and tissue engineering (Figure 1). Critically, iTx offer step-change improvements in efficacy compared with current standards of care (1) for a range of clinical indications and unmet therapeutic needs — particularly oncology. The clear efficacy of iTx is in contrast with some previous regenerative medicine approaches, including early mesenchymal stem cell (MSC) therapies…

April 14, 2015

Banking Cord Blood for Stem Cell Research

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Cord blood is becoming an increasingly popular and important topic of discussion among expectant parents. It comes from a newborn’s umbilical cord and contains hematopoietic stem cells (HSCs), which are the building blocks of a body’s blood and immune system. After a baby is born, cord blood is routinely discarded as medical waste — unless the parents choose to have what blood remains in the umbilical cord collected. Presently, more than 90% of cord blood is discarded, limiting the potential…

October 16, 2014

Transformative Healthcare: The National Biomarker Development Alliance

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A newly launched independent, nonprofit organization — the National Biomarker Development Alliance (NBDA) — will broadly engage leaders in industry, academia, patient groups, and government from across the United States. It was announced on 13 January 2013 at the National Press Club by the Research Collaboratory (RCASU) at Arizona State University (ASU). The mission of the NBDA is to address the complex and urgent challenge of creating standards needed for end-to-end, systems-based biomarker research and development (R&D). The alliance is…

June 1, 2014

Thinking Strategically

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At the recent Phacilitate Gene and Cell Therapy Conference (27–29 January 2014 in Washington, DC), BPI’s editor in chief Anne Montgomery and publisher Brian Caine spoke with Richard Grant, executive vice president, life sciences, at Invetech (www.invetech.com.au); and Brian Hanrahan, program manager at Invetech. They and their colleagues were instrumental in advising us how to bring ongoing discussions of regenerative medicines into BPI four years ago for the first Cell Therapy Supplement issue. We asked them to comment generally on…

March 1, 2014

Cell Therapy Bioprocessing Technologies and Indicators of Technological Convergence

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The cell therapy industry is undergoing a natural evolution from scientific curiosity into a commercially and clinically attractive opportunity (1). This evolution is by no means complete, and growing evidence suggests that its progression is driving significant developments in cell therapy bioprocessing — notably, convergence. Table 1:&#8 194; () Progressively, bioprocessing technologies primarily used in production of noncell-based products are being evaluated for cell therapy bioprocessing applications (2). Consequently, this process of convergence is leading to an increasing proportion of…

March 1, 2014

Effective Cryopreservation and Recovery of Human Regulatory T Cells

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The list of conditions being targeted by cell therapies is rapidly growing, but commercializing cells for widespread medical use will require standardized laboratory practices. Development processes must be adapted specifically for cell-based drug products. Regulatory T-cell therapy represents a promising new frontier in the immunotherapy of autoimmune disorders, especially for patients who have been refractory to available treatments. Because of intrinsic fragility, cell therapy products can be highly sensitive to variations in manufacturing procedures. Standardization of drug-product cryopreservation and storage…

March 1, 2014

Spray-Dry Manufacture of Vaccine Formulations

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Development of vaccines and immunotherapeutics has expanded rapidly due to technological advances in the fields of molecular biology and bioprocess engineering, as well as a smoothing of management and material logistics worldwide. Expression platforms and novel cell lines have enabled creation of increasingly complex vaccines. The advancement of vaccine formulation development is also capitalizing on new advances in manufacturing that use model-based methodologies gleaned from physiochemical principles, process analytical tools, and systematic approaches to problem solving. Herein we highlight recent…

October 1, 2013

Container–Closure Integrity

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An increasing number of biopharmaceuticals — including vaccines, stem cells, and proteins — require cold storage to maintain efficacy before use. However, the ability to maintain container–closure integrity (CCI) during cold storage is not completely understood. Concerns about CCI failure have been raised for storage and shipment of such products in rubber-stoppered vials under cold conditions (e.g., −80 °C or on dry ice). Commonly used butyl stoppers are believed to lose their elastic properties below their glass transition temperature (Tg),…

October 1, 2013

Moving Forward with a Gene Therapy for Damaged Hearts

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A cocktail of three specific genes can reprogram cells in the scars caused by heart attacks into functioning muscle cells. Adding a gene that stimulates the growth of blood vessels enhances that effect, say researchers from Weill Cornell Medical College, Baylor College of Medicine, and Stony Brook University Medical Center in a report that appears online in the Journal of the American Heart Association (1). “The idea of reprogramming scar tissue in the heart into functioning heart muscle was exciting,”…

September 1, 2013