Author Archives: Cheryl Scott

Spotlight: Niche-Disease — Barth Syndrome

Barth syndrome is a serious X-linked genetic disorder that primarily affects boys. It is caused by a mutation in the tafazzin gene (Taz) that creates an inborn error of lipid metabolism. The condition is named after Dutch pediatric neurologist Peter Barth, who published his discovery of it in 1983. The syndrome often manifests at birth in a number of ways. Patients are born hypotonic, show signs of cardiomyopathy within the first few months of life, and despite adequate nutrition experience…

To Serve and Promote: A Conversation with BIO’s President and CEO

BIO is the world’s largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products. BIO plays a leading role in shaping public policy related to the biotechnology industry — at the state, national, and international levels. Jim Greenwood has been BIO’s president and CEO for 10 years,…

BioProcess Theater: Analytical Services

BioProcess International launched about the same time as a major FDA regulatory announcement that has radically altered biopharmaceutical development: The quality by design (QbD) initiative is an important part of the agency’s 21st-century good manufacturing practice (GMP) approach, which is changing how regulators review product applications and thus how companies must approach them (1). It has placed increasing pressure on analytical laboratories, whose work is more important to the success of biotherapeutic products than ever before. Backed by harmonized tripartate…

BioProcess Theater: Cell-Line and Process Development

Process development forms the core of BioProcess International’s coverage and interests. From cell-line engineering through seed-train and production cultures, the results of which are harvested and purified to yield a bulk drug substance, every biologic therapy and vaccine begins its life at small scale in a research and development laboratory.Cell lines may be mammalian, insect, microbial, or even plant-sourced (1). They all require different types of culture media and supplements. And purification options are many and diverse. Process intermediates are…

BioProcess Theater: Formulation, Fill and Finish

Fragile proteins and other biomolecules need protection as stable drug products. The larger a molecule is, the more difficult it will be to make, ship/store, and administer to patients. Biotech drug formulators have many concerns to juggle in their work, beginning with the physicochemical characteristics of an active molecule and including the reliability, cost, and availability of analytical methods; the array of excipients and adjuvants on the market; evolving delivery methods and devices; patient preferences and behavior, as well as…

BioProcess Theater: Clinical and Commercial Manufacturing

When it comes to clinical and commercial manufacturing of therapeutic products, outsourcing is an integral part of the biopharmaceutical industry. In the 21st century, product sponsors are increasingly relying on expert contract assistance in process development and production of clinical and commercial materials. Many companies are reaching beyond their local and national borders to extend networks of partnerships into emerging markets, particularly in Asia (1, 2). Most biologics are proteins, with monoclonal antibodies (MAbs) dominating the scene over a number…

Spotlight

Niche-Disease: Pompe’s Disease by Cheryl Scott Pompe’s disease is a genetic disorder that causes a cellular buildup of the complex sugar glycogen. Its accumulation in organs and tissues impairs their ability to function normally. Since the disorder was identified in 1932 by Dutch pathologist J.C. Pompe, researchers have classified three forms of Pompe disease (also called glycogen storage disease type II or acid maltase deficiency): classic infantile-onset, nonclassic infantile-onset, and late-onset. The disease is inherited in an autosomal recessive pattern.…

QBD Manufacturing Strategies: The CMC Strategy Forum Series, Part 2

The CMC Strategy Forums provide a venue for biopharmaceutical product discussion. They focus on relevant chemistry, manufacturing, and controls (CMC) issues throughout the life cycle of a therapeutic and thereby foster collaborative technical and regulatory interaction. Forum chairs share information with regulatory agencies to help them merge good scientific and regulatory practices. Outcomes of the forum meetings are published in BioProcess International and on the CASSS website. This process is meant to help ensure that biopharmaceutical products manufactured with advancing…

Special Report: The Path to Vaccine Profitability

Managing vaccine supply chain improvements involves a complex interaction of laboratories, other facilities, CMOs, and suppliers. Since the business of making vaccines became a commercial proposition, profitability has often been elusive. The economics are difficult: Costs of development and production, already high, are rising. Profit margins historically have been lower than those of other pharmaceutical products, in part because of the complexities of manufacturing and distributing vaccines as well as their stringent safety, testing, and quality requirements.

Modern Laboratory Design: Creating a Space for Effective Collaboration

When asked to envision a modern biotechnology laboratory, lay persons might describe what they’ve seen on an episode of CSI: Miami. Gleaming glass and striking colored lights might look good on television, but they are not what biological researchers need to do their work most effectively. Most of the real biological laboratories I’ve visited, in fact, have been stark, white, fluorescent-lit environments that more resemble something out of 2001: A Space Odyssey. But those are becoming passé. The newest concepts…