This week’s headlines include: Moderna considering creating an mRNA monkeypox vaccine amid growing demand for shots, Amgen bets on ChemoCentryx’s rare disease drug in $3.7 billion deal, Global Blood Therapeutics Stock Soars Following Pfizer M&A Rumors, FDA considering dividing Jynneos doses into fifths to increase vaccine supply, U.S. Government Places $26 Million Order for Siga’s Monkeypox Drug, and Draft guidance: New options for submitting expedited safety reports for IND-exempt BA/BE studies.

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In Case You Missed It, Recent Articles on Cell Culture Dish and Downstream Column:

Scalable AAV Production Platform from Bench to Commercial Manufacturing

Gene therapies treat disease at a molecular level by transmitting genetic information to a target cell to correct a genetic defect, to inhibit the expression defective gene, to restore normal function or to impart a new function to the cell to treat or prevent disease. The clinical successes of genetic medicines have driven increased investment to implement these therapies for broader indications beyond rare diseases. Adeno-associated virus (AAV) is the vector of choice of many in vivo gene therapies and as more therapies reach the clinic, technological challenges must still be overcome to fully unlock its potential…

New Small Molecule Cocktail Provides Effective hPSC Cytoprotection to Increase Efficiency in Stem Cell Workflows

Human pluripotent stem cells (hPSCs) have an extensive capacity for self-renewal and the ability to differentiate into virtually any cell type in the body. These cells represent an exciting paradigm for disease modeling, drug discovery and regenerative medicine.  However, a significant challenge to the clinical translation of hPSCs is the inherent sensitivity of the cells to their in vitro environment, where poor survival during both routine culture and cell manipulations, especially involving single-cell dissociation, considerably limits the efficiency and scalability of this promising technology…

CGT leaders bluebird bio, BMS and Janssen share industry predictions for the decade to help you reinforce your go-to-market strategy

Despite the novelty of the CGT space, it’s already sending shockwaves through pharma. But getting into the mind of industry trailblazers can be a hard task for those trying to assemble an infallible launch strategy…

Accelerating cell and gene therapy development using custom media solutions early in process development

In this podcast, we spoke with Dr. Brad Taylor from Nucleus Biologics and Randy Jacinto from Cytiva about the benefits of using a custom media for cell and gene therapy process development. Topics included current development challenges, increases in the number of cell and gene therapies in clinical trials, and employing custom cell culture media solutions to address these issues…

Achieve unparalleled structural elucidation and sensitivity with the ZenoTOF 7600 system from SCIEX

Liquid chromatography with tandem mass spectrometry (LC-MS/MS) is one of the most versatile and high-performance analytical tools for the detection, characterization and quantification of biotherapeutic molecules and vaccines. However, emerging modalities require new analytical methodologies to enable in-depth characterization and mitigate potential risks early in the development process. In addition, the increasing potency of next-generation biologics drives the need for more sensitive assays for bioanalysis studies. Uncovering new perspectives for molecules, which might have even been previously undetectable, requires overcoming the specificity and sensitivity limitations of current LC-MS/MS technologies. In response to these needs, SCIEX has launched the ZenoTOF 7600 system. This accurate mass, quadrupole time-of-flight (QTOF) system can improve compound identification in protein, oligonucleotide, metabolite and lipid analyses, and it can help scientists uncover new insights and drive decision making processes faster. In addition to offering collision-induced dissociation (CID), the instrument offers a new tunable fragmentation technology based on electrons. The tunable electron activated dissociation (EAD) technology tackles the elucidation of challenging molecules across all charge states, allowing for new depths in the characterization of molecules…

Biomanufacturing Quality Management Systems – A holistic approach

In this podcast, we interviewed Katie Keller, Director of Quality and Safety at Asahi Kasei Bioprocess America, about the importance of quality management and how to achieve the best possible results. Topics included the most critical elements of quality management, how to ensure the purchase of high-quality equipment, and future trends…

Flexible single-use mixers are key to achieving a next level mixing experience and reducing delivery lead times

In this podcast we spoke with Klaus Kienle, Global Product Manager for the Mixing portfolio at Pall Corporation about the latest mixing technologies including single-use solutions, the need for increased flexibility, and a more standard vendor agnostic approach…

Mustang Membrane Technology for AAV Empty and Full Capsid Separation

Interest in Adeno-Associated Virus (AAV) purification approaches have been increasing with recent AAV based gene therapy regulatory approvals. Purification approaches that have been developed for monoclonal antibodies and other recombinant proteins have been modified for the purification of viruses. However, as the demand for gene therapy increases, so does the need for the industry to create a fit-for-purpose purification solution. Resins, which are a mainstay of protein purification, can’t fully address virus purification because of diffusion limitation: The pores are often so small that they exclude viruses, limiting the binding capacity and requiring longer residence times. This, in turn, means operating at lower flow rates and increasing processing times…

Single-Use in Biotech – Addressing Opportunities and Challenges

Over the past decade, the growth and adoption of single-use technologies in biomanufacturing has been steep and sustained. What began primarily with single use bioreactors has expanded to include single-use components from early upstream processes through downstream and into commercial manufacturing. As of 2018, single-use systems were being used for about 85% of preclinical and clinical biomanufacturing and are increasingly being incorporated into commercial manufacturing¹. The benefits of single-use technologies have been well documented and include risk mitigation, increased efficiency, reduced personnel requirements, less validation time, and customizable solutions. The technology has also been found to reduce utility and water costs for cleaning and sterilization, as well as lower batch changeover time. This is particularly important for facilities that run smaller manufacturing lots, with several different products or are looking to maximize facility flexibility…

Full Adeno Associated Virus (AAV) Capsid Enrichment Using Mustang® Q Membrane

Adeno-associated virus (AAV) vectors are becoming increasingly important gene delivery platforms for the development of gene therapies for several reasons. They are relatively safe with low immunogenicity, and demonstrate high gene transfer efficiency and stable long-term expression in both dividing and quiescent cells. As well, a number of AAV serotypes exist that differ in their tissue tropism, or the types of cells they infect, making AAV a very useful system for transducing specific cell types. Typically, AAV production occurs within a packaging cell line, such as HEK293T cells, where plasmid containing the gene of interest (GOI) are assembled into a protein capsid shell. A current challenge in manufacturing is that the encapsidation efficiency for AAV is highly variable, which produces a heterogeneous mixture of AAV particles that contain the GOI (full) and particles without any genome (empty) or with fragmented, non-functional DNA (partial). Because only the full AAV capsids can exert a therapeutic effect in vivo, the other capsids are viewed as a process impurity and removing them is a necessary step in downstream purification process to achieve a consistent drug product. Existing methods to purify full AAV capsids like analytical ultracentrifugation (AUC) are effective yet have limitations in throughput and cost for a manufacturing environment, which has driven the development of more scalable methodologies for AAV purification…

Report:

A recent white paper, Osmolality monitoring for UF/DF and CEX capture of mAbs from mammalian cell cultures, published in collaboration with Advanced Instruments examined how osmolality can be used within downstream processing as an additional critical process parameter.

The goal of the study was to understand the impact of monitoring osmolality during an Ultrafiltration/Diafiltration (UF/DF) step prior to CEX capture of the monoclonal antibody Rituximab. The process of UF/DF in downstream workflows is essential within the production of biological therapeutics, such as monoclonal antibodies, proteins (e.g. hormones) and nucleic acids. The UF/DF steps can be undertaken separately or in combination to prepare the target molecule, including sample cleanup, purification, concentration, buffer exchange, and desalting. Since both pH and conductivity are both standard metrics used in downstream process analytics, they were used as a base to then build a model around osmolality measurements within the process of UF/DF.

Download the full white paper here: Osmolality monitoring for UF/DF and CEX capture of mAbs from mammalian cell cultures

Headlines:

“Moderna considering creating an mRNA monkeypox vaccine amid growing demand for shots,” ABC News

Amid growing concerns over the potential threat of monkeypox, executives from Moderna said Wednesday they have initiated a research program to consider whether the company could create a monkeypox vaccine with mRNA technology. “We’re obviously very aware of the monkeypox concern and obviously very sensitive to recent announcements,” Moderna President Stephen Hoge said during an investor call…

“Amgen bets on ChemoCentryx’s rare disease drug in $3.7 billion deal,” Reuters

Amgen Inc on Thursday agreed to buy ChemoCentryx Inc (CCXI.O) for $3.7 billion to gain access to a potential blockbuster treatment for inflammatory disorders and beat quarterly revenue estimates on demand for its bone disease drug. The deal, announced before markets opened, will also hand the company control of at least two experimental therapies for immune disorders, a field in the spotlight following AstraZeneca’s 2020 purchase of Alexion Pharmaceuticals for $39 billion.

“Global Blood Therapeutics Stock Soars Following Pfizer M&A Rumors,” BioSpace

Shares of Global Blood Therapeutics are surging once again after reports indicate Pfizer is the mystery suitor for the company. The stock has skyrocketed more than 38% to $66.61 per share following reports that the pharma giant could plunk down $5 billion for the sickle cell disease-focused company. Firstreportedby The Wall Street Journal, Pfizer is looking for a quick buy of the Bay Area-based company. However, there could possibly be other suitors in the mix, the Journal said.

“FDA considering dividing Jynneos doses into fifths to increase vaccine supply,” The Hill

The Biden administration is considering splitting doses of the smallpox vaccine, which are being used to prevent monkeypox amid the current outbreak, into five smaller doses, the head of the Food and Drug Administration (FDA) said Thursday…

“U.S. Government Places $26 Million Order for Siga’s Monkeypox Drug,” FDA News

The U.S. government’s Biomedical Advanced Research and Development Authority (BARDA) has placed a $26 million order for Siga Technologies’ intravenous Tpoxx treatment for monkeypox infections. The intravenous drug offers an alternative treatment option for patients who have difficulty swallowing the oral formulation of the drug.

“Draft guidance: New options for submitting expedited safety reports for IND-exempt BA/BE studies,” Regulatory Focus

The US Food and Drug Administration (FDA) has issued draft guidance providing instructions for how generic drug manufacturers can electronically submit expedited individual case safety reports (ICSRs) for serious adverse events through the FDA Adverse Event Reporting System (FAERS)…