Manufacturing

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…

Sharing Viral Vector Expertise: A Conversation with Yposkesi’s Chief Executive Officer

As a full-service contract development and manufacturing organization (CDMO) specializing in gene therapy development, Yposkesi produces recombinant adenoassociated virus (AAV) and lentivirus (LV) vectors using adherent-based and suspension-adapted cell expression platforms. Alain Lamproye joined the company as chief executive officer (CEO) in January 2017, having served previously as president of the biopharmaceutical business unit of Novasep (2012–2017) and as CEO of Henogen, its subsidiary dedicated to gene therapy. He has held managerial positions in pharmaceutical operations at Merck Serono (including…

Ask the Expert: Key Considerations for Cryogenic Preservation and T-Cell Viability

Cryopreservation provides critical protection for cell therapies by minimizing genetic changes. But cooling too slowly or quickly risks diminishing cell viability upon thaw. On 11 March 2020, Peter Kilbride (senior research scientist) and Julie Meneghel (cryobiologist), both of Cytiva (formerly GE Healthcare Life Sciences), discussed the importance of controlled-rate cryopreservation. Illustrating how mammalian cells change when frozen, Kilbride and Meneghel offered concrete cryopreservation strategies and identified temperatures at which it is safe to stop controlled cooling and transfer drug product…

A Healthy Biosimilars Market Promotes Innovation and Affordability

Innovative drug manufacturers require an opportunity to recoup capital and opportunity costs that they incurred to develop new medicines. Once patents have expired, competitors should be empowered to promote widespread affordability. An exclusivity period followed by a competitive market would promote the otherwise incompatible objectives of incenting innovation and promoting affordability. That careful balance exists for small-molecule medicines but not yet for biologics. The innovation side of the biologics market is working as intended, but a robust market for lower-cost…

eBook: Next-Generation Vaccines — COVID-19 Challenges, Opportunities, and Patent Questions

Resolving the COVID-19 pandemic depends on treatments, testing, and ultimately a widely disseminated vaccine against SARS-CoV-2. In recent decades, the biopharmaceutical industry has developed new approaches to vaccination using antigens, virus-like particles (VLPs), viral and bacterial vectors, and nucleic acids. Current events have placed those innovations at the front and center of public attention, offering many companies an opportunity to demonstrate their potential in an unprecedented way. Here, BPI’s senior technical editor describes the challenges that developers face in doing…

Developing Process Control Strategies for Continuous Bioprocesses

Process control enables biomanufacturers to ensure that operating parameters are within defined specifications. A control strategy should be established during early stages of process development while process and product performance are being defined using risk-based methods such as quality by design (QbD) and process analytical technologies (PATs). Confirming process control as an essential part of product development creates greater process knowledge and understanding and provides the first steps toward process optimization. By understanding how process performance relates to product quality,…

eBook: A Light-Chain Platform for Developing Bispecific Antibodies

Biopharmaceutical researchers are focusing on novel platforms to quickly develop bispecific antibody (BsAb) therapies for treating complex diseases such as cancer. BsAb therapies offer several advantages because they are designed to bind two unique epitopes. In this report, Bill Lundberg, president and chief executive officer at Merus, describes his company’s efficient approach to developing BsAbs using “common light chain” technology and other proprietary strategies. Lundberg also addresses the company’s approach to typical BsAb development and manufacturing challenges. Fill out the…