Cell/Gene Therapies

eBook: Cell Therapy — Lessons Learned from Working with Vectors and Cells

Gene-modified cell therapies hold much promise for cancer treatment. Currently, the most popular approach leverages T cells expressing chimeric antigen receptors (CARs), which give immune cells the specificity needed to bind with and destroy malignancies. Despite rapid progress in drug discovery and development, the biopharmaceutical industry still has much to learn about manufacturing CAR T-cell therapies in commercially feasible ways. In this eBook, BPI’s managing editor speaks with subject-matter experts from VIVEbiotech, Castle Creek Biosciences, and Bristol Myers Squibb (BMS)…

Cell Therapy — Supply Chain Discussions

The 2017 approval of Kymriah (tisagenlecleucel) has paved the way for other cell therapy products to reach the patients who need them. Each passing year promises to open the regulatory floodgates to more product approvals, but technical and manufacturing issues continue to keep initially high expectations from being realized. One barrier is supply chain complexity, especially for the subset of autologous cell therapies. This eBook features expert commentary from Be The Match BioTherapies, a company providing more than 50 product…

Improving Scalability of AAV and Lentivirus Production: Tailored Transfection Reagents Enable Large-Scale Manufacturing

Scientific advances have increased the safety and efficacy of recombinant viral-vector technology for cell and gene therapies. In this special report, we will discuss how tailored transfection reagents improve the scalability of AAV and lentivirus production. We will also focus on the challenges of scaling up viral vector production platforms and the importance of selecting the right transfection reagent for this process to increase the titer and quality of viral production to generate more therapeutic doses per run. Fill out…

Purity by Design

After launching Nereus LentiHERO, a game changing fit-for-purpose solution for lentiviral vector purification in 2022, Astrea Bioseparations present this end of year report with a selection of interviews, application notes, and articles. Identifying the gaps in lentiviral purification, they discuss how to increase processing efficiency, purity, and recovery of LVV particles. They also look towards the future to bring a radical change in bioprocessing for cell and gene therapies. The Nereus LentiHERO spin column is the first product in a…

Allogeneic Cell Therapy Manufacturing: Preparing for Tomorrow’s Success

Cell therapies are promising new drug products that treat or cure diseases that, until the past decade, had no other treatment options. Several autologous cell therapies have been approved, and their efficacy has been proven, especially in immunooncology. However, autologous therapies can present some difficulties for both developers and patients (e.g., short timelines, point-of-care drug administration). Allogeneic cell therapies are not associated with those challenges. For example, patient access to an autologous treatment can take months, time that patients with…

Addressing Challenges in Analyzing Heterogeneous AAV Populations

Adenoassociated virus (AAV) vectors are made from nonenveloped virus capsids that contain single-stranded DNA. As a leading delivery system for gene therapy, AAVs are in development to treat a number of genetic diseases (1). As the industry has advanced, the number of clinical trials involving such vectors has risen dramatically (2), increasing the need for effective manufacturing and quality control (QC) methods. During biomanufacturing, expressed AAV capsids can incorporate both target and host-cell DNA in a heterogeneous population. Viral capsids…

eBook: Cryopreservation — Special Considerations for Cell Therapies

Freezing is an established approach to maintaining bulk drug substances/products for several biopharmaceutical modalities. Regardless of product type, a cryopreservation process requires extensive testing, monitoring, and control of freezing and thawing conditions. However, because living cells are significantly larger and more complex than monoclonal antibodies (MAbs) and other protein-based biologics, cell therapies raise distinctive concerns for cryopreservation processes. In this eBook, BPI’s managing editor speaks with Erik Wood (cofounder, chief science officer, and senior vice president of Ossium Health) to…

Harnessing the Untapped Power of the Human Commensal Virome

Viruses are ubiquitous members of every ecosystem, including the human body (1). A common misconception is that all viruses are harmful. In fact, their relationships to human hosts can be pathogenetic, symbiotic, or commensal. In the latter case, a virus benefits from but neither harms nor helps its host (2). Commensal viruses are as abundant as human cells, and their coexistence has persisted for millennia, permitting our immune systems to recognize them as part of us. Among the most abundant…

Optimize Your Early Preclinical Process for Gene Therapy Success

Early decisions can impact the success of a novel gene therapy, so how can therapeutics companies make sure to set off on the right track? In this article, drug developers will learn some things that they should consider at the start of their therapeutic journey. We explore the impact of early technical decisions, including the importance of seeking high-yielding plasmids and a well-validated cell line. We cover logistical considerations, such as plasmid supply chain challenges and how to avoid them.…

Addressing Unwanted Immunogenicity in Gene Therapies

Immunogenicity is the ability of a substance, such as a foreign and/or potentially dangerous protein, to provoke an antigen-specific immune response. However, some immune responses can be detrimental, such as in autoimmune diseases and unwanted reactions to biological therapeutics. The latter case can compromise biopharmaceutical safety and efficacy, and preexisting immunity against biologic components can preclude patients from receiving life-changing treatments, perhaps most notably in gene therapy (1). Gene therapies are designed to target the root cause of a genetic…