Cell/Gene Therapies

Future Supply-Chain Needs for Allogeneic Cell Therapies: Why Strategic Partnerships Are Critical

Allogeneic products are an attractive option for cell-therapy developers because multiple batches can be manufactured using apheresis material collected from one healthy donor — and because the resulting therapies could be made available as off-the-shelf products. The appeal of this approach is apparent from growth in allogeneic-therapy development. According to the Alliance for Regenerative Medicine, the number of clinical trials for allogeneic cell-based cancer treatments has increased by 30% over the past five years. Early in 2022, allogeneic candidates accounted…

Raman Spectrometric PAT Models: Successful Transfer from Minibioreactors to Larger-Scale, Stirred-Tank Bioreactors

Spectroscopic sensors are powerful tools for bioprocess monitoring within the process analytical technology (PAT) initiative of the US Food and Drug Administration (FDA). The PAT framework includes process understanding based on scientific background with the aim of monitoring and controlling critical process parameters (CPPs) that influence critical quality attributes (CQAs) of final biological products. The driving force for PAT implementation is a need to realize consistent product quality, process intensification, and real-time manufacturing control (1, 2). Using real-time spectroscopic measurements…

Deriving Mesenchymal Stromal Cells from Umbilical Cord Lining and Wharton’s Jelly: A Comparative Study of Extraction Methods and Culture Media

Mesenchymal stromal cells (MSCs) are multipotent, self-renewing progenitor cells that can differentiate into adipocytes, chondrocytes, and osteocytes (1). Cultured MSCs are plastic-adherent and spindle-shaped, and they express cell-surface markers CD44, CD73, CD90, and CD105, but not CD14, CD34, CD45, CD11b, CD79a, CD19, or HLA-DR (2, 3). First isolated from bone marrow (BM), human MSCs have been investigated extensively in clinical studies. MSCs also have been isolated from adipose tissue (4) and peripheral blood (5). Perinatal organs and tissues such as…

Overcoming Challenges in Viral Vector Production for Gene Therapy Using HEK Cell Cultures

An estimated 300 million people worldwide live with rare diseases, and over 70% of such disorders are caused by genetic mutations (1, 2). Cell and gene therapies offer hope and potential cures for many previously untreatable diseases. Accordingly, the global gene therapy market is expected to be worth USD 5.02 billion by 2028, a significant growth from USD 1.46 billion in 2020 (3). Manufacturing gene therapies will be a key challenge over the next two decades. But just a few…

Bringing Gene-Therapy Product Quality Into Focus

Regulatory agencies are scrutinizing gene-therapy product quality more closely than ever, yet such therapies still are produced in small batches and at high costs. Thus, drug companies are struggling to make safe and efficacious gene therapies available to patients. In an April 2022 presentation, Tim Kelly (chief executive officer of Oxford Biomedica Solutions, OXB Solutions) emphasized the importance of addressing both process output and product quality when manufacturing adenoassociated virus (AAV)-based gene therapies. Such an approach requires deep expertise and…

Improving Viral Vector Manufacturing: Modeling Costs to Help Optimize Processes

Manufacturing costs remain high for gene therapies delivered by adenoassociated virus (AAV) vectors. The biopharmaceutical industry must minimize such expenses because they account for significant proportions of the high prices that patients pay for treatment. During a June 2022 webinar, Emmanuelle Cameau (leader for cell and gene therapy strategic technology partnerships at Pall Corporation) joined Maxime Dumont (cell and gene therapy product manager at webcast sponsor Polyplus-transfection) to describe their companies’ efforts to model AAV manufacturing costs. Cameau and Dumont…

Mass Photometric Analysis of Adenoassociated Virus Capsids

Current production processes for gene therapies based on adenoassociated virus (AAV) vectors generate many empty capsids. That problem complicates vector purification and diminishes product safety and quality. In a June 2022 webinar, Gareth Rogers (product manager at Refeyn Ltd.) observed that developers could benefit significantly from analytical instruments that assess empty-to-full (E:F) capsid ratios rapidly. He explained how the SamuxMP mass photometry system (Photo 1) could address such needs. Kirsty McManus (senior scientist in AAV characterization at Pharmaron Gene Therapy)…

BioProcess Insider Interview: Tom VanCott, Catalent

Moderator: Dan Stanton, Co-founder and Editor, BioProcess Insider. Featuring: Tom VanCott, PhD, Global Head of Product Development, Catalent. Contract development and manufacturing organization (CDMO) Catalent has expanded significantly over the past decade through strategic acquisitions and investments in cell and gene therapy (CGT) capabilities. VanCott reflected on how his company has supported such growth while manufacturing SARS-CoV-2 vaccines and therapeutics. Catalent was able to accommodate the sudden and extraordinary demand for COVID-19 products because it already had been increasing capacity…

BioProcess Insider Interview: Claudia Melara, Gamma Biosciences

Moderator: Dan Stanton, Co-founder and Editor, BioProcess Insider. Featuring: Claudia Melara, Vice President of Key Accounts, Gamma Biosciences. Since its launch in 2020, Gamma Biosciences has acquired or purchased controlling stakes in several solutions providers, the capabilities of which now span the entire biomanufacturing spectrum. Gamma Bio helps drug developers move their products from preclinical research through good manufacturing practice (GMP) production. To spur on commercialization of advanced therapies, Gamma Bio now has established a key accounts program. Drug developers…

Cell and Gene Therapy Modalities: Business and Manufacturing Strategies Influencing the Decision to Develop One Therapy Type Instead of Another

Moderator Patricia Seymour opened the panel by asking each presenter to introduce himself and his company’s technology. Caribou Biosciences: Cell Therapies Panelist Justin Skoble (vice president of technical operations with Caribou Biosciences) is responsible for developing the company’s next-generation CRISPR genome-edited cell therapies. Before joining Caribou in 2019, he was chief development officer at Acton Therapeutics and served in positions of increasing responsibility in immuno-oncology and vaccine development at Aduro Biotech, Anza Therapeutics, and Syros. His experience encompasses development of…