Cell/Gene Therapies

Where Will Technology Take Cell Therapy?

    The cell therapy industry’s biggest challenge is in manufacturing. Technologies are needed to support expansion of large numbers of cells for commercial production. A number of sources are presenting options: e.g., standard two-dimensional tissue cultures that “grow up” to Corning HYPERFlask and CellSTACK or Nunc Cell Factory systems; hollow-fiber–based equipment; and disposable bags and traditional stirred-tank bioreactors. Each has its place and application, but how can companies choose among them? Where and when do they initiate scale-up process…

Cell Therapy Bioprocessing

          The past 15 years have seen approval and commercialization of the first cell-based therapeutics, including cartilage repair products; tissue-engineered skin; and the first personalized, cellular immunotherapy for cancer. Those successes are outnumbered, however, by all too common product failures. Notable failures can be attributed to commercial concerns such as high cost of goods (CoGs) and technical hurdles such as inadequate characterization, high process variability, and loss of product efficacy when manufacturing is scaled up (1).…

Meeting the Challenges in Manufacturing Autologous Cellular Therapies

Personalized medicine is a promising new approach to disease treatment. The ultimate in personalized medicine is a cellular therapy manufactured specifically for an individual patient using his or her own cells. But this autologous approach to generating immunotherapies has unique manufacturing challenges. Each patient receives an individual product batch, which needs to be manufactured, tested, and released. So thousands to tens of thousands of batches could be made for each indication every year. Given the personalized nature of these therapies,…

Industry Roundtable

    With one eye on commercialization and the other on monitoring every-day challenges, cell therapy manufacturers are asking critical questions about process efficiency, ensuring quality, and satisfying regulatory demands. In this “virtual” roundtable discussion (participants were asked questions separately), cell therapy industry representatives answer key questions in hopes of broadening understanding about this new class of biopharmaceuticals. Participants in this roundtable are Timothy Fong, PhD (director cell therapy, Becton Dickinson Biosciences), Annemarie Moseley, PhD, MD (CEO, Repair Technologies), Firman…

Addressing Business Models, Reimbursement, and Cost of Goods

The early ISCT organization provided a powerful forum for sharing solutions, developing standards, and moving the emerging concepts in cell therapy forward as the field grew up and out of academia. Currently, the ISCT organization is uniquely positioned to facilitate sharing of best practices, standards, and strategies across the for- profit cell therapy industry through its Commercialization committee. The Business Models, Reimbursement and CoGS (cost of goods sold) subcommittee of the ISCT Commercialization committee was formed to address several key…

Industry Educational Platforms Drive Commercialization Objectives

    Within the International Society for Cellular Therapy’s (ISCT’s) Industry Commercialization committee, Tracey Lodie, director of immunology and stem cell biology at Genzyme, chairs the Industry Education subcommittee, which was established in May 2010. In an interview with BPI, she described the subcommittee’s objectives and how they tie into the manufacturing, testing, and commercialization challenges for cellular therapies.   Reducing the Risk   “ISCT is working toward becoming an informational hub, acting as a resource to de-risk cell therapy…

Building from the Ground Up

    New treatment modalities — as transformative as they may be of our approaches to human healthcare — still need to be profitable for their developers, provide the sorts of returns desired by investors, and be accessible to patients financially. As many industry experts have told us, the venture capital climate these days is much different from that of the early, giddier days of monoclonal antibodies. And with criteria still-emerging around the world for how regenerative medicines are and…

Working Together for the Future

Most individuals who choose to pursue a career in healthcare would say they do so because they are driven by a fundamental desire to help people. If you ask people why they decided to work in the field of regenerative medicine, many will tell you it’s because they believe it is the most exciting area of medical research and that it holds the greatest potential to transform medicine as we know it. The transformational potential of stem cells and regenerative…

Building a Bridge to Commercial Success

The history of the biopharmaceutical industry is one of continual invention and reinvention, of business models that have adapted to weather uncertain product futures and shifting economic fortunes. Some of us followed the up-and-down (and often financially painful) progress of monoclonal antibodies toward their eventual commercial success — a wealth of experience to draw from as other classes of products make their way from laboratories and onto the market. The vast majority of regenerative medicines are still produced at laboratory…

Therapies of Tomorrow Require More Than Factories from the Past

Live cells are being incorporated as active agents and delivery vehicles for a broad range of emerging therapeutic strategies. Successful commercialization of a cell therapy requires more than proving its safety and efficacy to regulators. Ultimately a therapy must be commercially viable, allowing enough patients to be treated with an adequate financial margin to justify investment in it as a product. “Whether the cells used are universal (allogeneic) or patient-specific (autologous), it is unlikely to be wholly one or the…