Cell/Gene Therapies

eBook: Viral Vector Purification —
A Discussion of Current Challenges and Methods

Adenoassociated viral (AAV) vectors have become synonymous with gene therapy delivery. However, because they are produced in such small quantities and because their upstream processes carry comparatively large amounts of host-cell DNA and other impurities, AAV purification can be challenging. Several researchers have applied different chromatographic strategies, but no universal method has been adopted in the biopharmaceutical industry. This eBook features a discussion among several industry experts that explores challenges specific to AAV purification, shedding light on whether current strategies…

eBook: Trends in Facility Design —
In-House Manufacturing Considerations for Cell and Gene Therapy Production

Manufacturing and facility challenges facing cell and gene therapy companies are similar to but more complex than those encountered by companies that produce traditional biopharmaceuticals such as vaccines, monoclonal antibodies, and other therapeutic proteins. A single product can have multiple components, manufacturing of which may or may not be outsourced. Project timelines are short, production technologies are new and evolving, and clinical demands change rapidly. Increasing competition for contract manufacturing services requires reserving capacity far in advance, which in most…

Developing Advanced-Therapy Products Through Global CDMOs

Tremendous growth in the cell and gene therapy (CGT) industry is driving unprecedented demand for manufacturing services. To be sure, advanced-therapy developers increasingly are choosing to install in-house capabilities. Doing so can offer companies greater control of their processes, timelines, and budgets than they might have when outsourcing products (1). But industry experts agree that contract development and manufacturing organizations (CDMOs) will remain integral to CGT manufacturing and commercialization (1, 2), especially with veteran contract partners scrambling to acquire CGT…

The Role of New Technology in Enabling Cell and Gene Therapies

Moderator Doug Miller, with Tim Kelly, Daniella Kranjac, Andy Ramelmeier, and Ran Zheng This part of the BPI Theater program focused on emerging bioprocess technologies and their implications for cell and gene therapy (CGT) products. Doug Miller assembled a panel showcasing a wide range of technical expertise and representing several CGT industry stakeholders. That pluralism yielded far-reaching discussions about what CGT capabilities exist, what advances could facilitate therapy manufacture, and how companies might pursue technological development. Viral-Vector Therapies Miller kicked…

Viral-Vectored Gene Therapies: Harnessing Their Potential Through Scalable, Reproducible Manufacturing Processes

We might not associate the jazz queen Ella Fitzgerald with 21st-century gene-based therapies, but the First Lady of Song was on to something back in 1939 when she sang “’T’Ain’t What You Do (It’s the Way That You Do It).” Although demonstrating the safety and efficacy of gene-based therapies in rigorous clinical trials is essential for gaining product approval from regulators, doing the bare minimum is insufficient. The way that such products are produced also matters. Manufacturing processes and protocols…

Fluorescent Nanosensors: Real-Time Biochemical Measurement for Cell and Gene Therapies

Cell and gene therapies are destined to transform the methods by which global healthcare challenges are approached and overcome (1). The US Food and Drug Administration is reviewing and approving an increasing number of cell and gene therapy products (2), and biopharmaceutical developers are dedicating immense resources to realizing the enormous potential of these therapeutics. Therefore, technologies that facilitate their effective and efficient manufacture will accelerate cell and gene therapies’ transition from medicines of the future to medicines of the…

Gene Therapy Trends and Future Prospects

Gene therapy is defined as the transfer of genetic information to a patient for treatment of a disease. Clinical investigation of such therapies began in 1990 with a treatment for a rare immunodeficiency disorder and since has expanded to almost 1,000 clinical studies in 2019 (1, 2). In its most straightforward incarnation, the goal of gene therapy for genetic diseases is long-term expression of a transferred gene at levels that are high enough to be therapeutic, an approach sometimes called…

Cell and Gene Therapies Get a Reality Check: A Conversation with Anthony Davies of Dark Horse Consulting Group

As founder of cell and gene therapy (CGT) specialist firm Dark Horse Consulting Group in California, Anthony Davies speaks from a quarter century of experience including former positions at Onyx Pharmaceuticals, Syrrx, ZymeQuest, Serologicals, Geron Corporation, Capricor, and 4D Molecular Therapeutics — and he currently serves on the board of directors for TrakCel and the scientific advisory boards for Akron Biotech and BioLife Solutions. In his plenary address at the Phacilitate 2020 Leaders World conference (part of Advanced Therapies Week…

Quality By Design for Advanced Therapies: An Informed Route to Enhanced Late-Stage Clinical Success and Empowered Process Flexibility

As advanced therapies, including regenerative medicines, progress toward commercialization and market approval, early warnings from key opinion leaders (1, 2) regarding the importance of better understanding quality target product profiles (QTPPs) and critical quality attributes (CQAs) of such products have resounded ever louder (see the “Terminology” box for definitions). Costly late-stage delays, redirections, and even abandonment of clinical programs can be linked to quality issues associated with inadequate understanding of process and product. Therefore, a review of the benefits of…

Transforming Personalized Medicine into Off-the-Shelf Cell Therapies

Initial progress in cell and gene therapy has seen 12 advanced therapeutic medicinal products (ATMPs) become available on the market in 2019 for a range of conditions, from monogenic diseases to cancer. Despite such progress, development of clinically and commercially successful cell therapies presents manufacturability challenges and questions about bypassing patients’ immune systems. The availability of rapid sequencing and next-generation bioinformatics has made it possible to understand the mechanisms of disease better and accelerate development of therapeutic responses. The same…