Author Archives: Maribel Rios

Preparing for Process Improvements: Discussions of the Cell Therapy Industry’s Supply Chain, Automation, and Control Needs

As editors, we are fortunate to have the opportunity to listen to biopharmaceutical developers and innovators discuss the intricacies of their work. Typically, we find that the best discussions come from asking two fundamental questions: What need did you observe in the industry that drove your work, and what technologies would help you do your job better? Over the past five years or so, the answers have shifted. Cell and gene therapy (CGT) innovators are focusing on increasingly complex diseases…

Automation of Potency Assays: A Strategic Journey

Cell-based potency testing provides quantitative data concerning a drug’s biological activity. Thus, it plays an essential role in biopharmaceutical quality control (QC), good manufacturing practice (GMP) product release, comparability determination, and stability testing for both drug products and drug substances. Potency is a critical quality attribute (CQA) often scrutinized by regulators and reviewers. Test methods are specific to a drug’s mechanism of action (MoA) and should be validated to internationally harmonized regulatory standards (1). The options preclude applying a simple…

Optimization of Processes and Advanced Platforms for Viral Vector Processing

Viral vectors are synonymous with gene therapies, so their development, production, and processing are of upmost importance to all gene therapy researchers and manufacturers. Every year, I look forward to attending the Cell and Gene Bioprocessing and Commercialization conference in Boston and talking to leaders in industry and academia about their current approaches to advancing gene therapies. Like most other meetings this year, the conference was entirely online and had to provide a shortened agenda. Nonetheless, there was no shortage…

November–December 2020:
From the Editors

As 2020 draws to a close, we can’t help looking back at this year in which the biopharmaceutical industry has received more attention than ever before — scientifically, politically, and otherwise. We editors always have been conscious of (and pretty good at) separating our work and home lives. As many of you have learned this year, that’s an important part of working from home, which we’ve been doing for over a decade. But in recent months, reading the news and…

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…

Optimizing Cell Line Development for High-Quality Biologics

For a host-cell system to generate high yields of recombinant proteins and other entities, cells must be derived from optimized and stable cell lines. However, cell line development (CLD) can be tedious and time-consuming work, and every stage in the CLD workflow has its limitations and challenges. Researchers are creating advanced strategies and tools to overcome those challenges, especially for complex biologics such as bispecific antibodies (BsAbs) and difficult-to-express (DTE) proteins. Online presentations from the CLD track of the BioProcess…

Plant-Cell Cultures and Cell Lines for Recombinant Protein Expression

Cell cultures derived from mammalian and bacterial cell lines are the conventional production systems in bioprocessing. But they also have their limitations. Media for mammalian cultures in particular are notoriously expensive, and traditional cell cultures can be highly sensitive to growing conditions. During the late 1980s and into the 1990s, plants and plant-derived cell cultures were introduced as alternative cell-culture systems (1, 2). Although transgenic plants (genetically modified) once looked promising in the early 2000s, the cost and manufacturing complexity…

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…

Scalable Manufacturing of Lentiviral Gene Therapies

Lysosomal storage diseases are caused by mutated genes that express defective lysosomal proteins, such as essential enzymes. For example, cystinosis is a metabolic disease caused by a defective gene that encodes cystinosin, an exporter protein (Figure 1). Avrobio develops gene therapies to treat lysosomal storage diseases. On 8 October 2019, the company announced that its first patient had been dosed in the AVR-RD-04 investigational gene therapy program, which involves genetic modification of the patient’s own hematopoietic stem cells to treat cystinosis.…