This week’s headlines include: Scientists to test tailor-made vaccine tech to fight epidemics, Orchard Therapeutics to Build Out Gene Therapy Manufacturing Site in Fremont, CA, Moderna Raises $604M and Nails Down Biotech’s Biggest IPO Ever, Roche’s lung cancer combo treatment wins FDA approval, U.S.-EU Partnership on GMP Inspections Adds Five Countries, and CRISPR controls obesity in mice by amplifying rather than editing genes,” FierceBiotech

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

Key Considerations in Gene Therapy Manufacturing for Commercialization – A panel discussion

This podcast is a recording from the panel discussion that I moderated at this year’s Cell and Gene Therapy Bioprocessing and Commercialization Conference. The discussion covers the latest in gene therapy manufacturing strategies, analytical analysis, cost of goods and key regulatory considerations…

Scalable Viral Vector Upstream Process for AAV Gene Therapy Manufacturing

Gene therapy is an exciting area of medicine that has the potential to treat a wide range of diseases for which no other treatments are available. One of the key components of successful gene therapy manufacturing is the production of the viral vectors that are used to deliver the gene of interest. Viral vector systems are by far the most widely used methods to deliver therapeutic gene products because of their infectious nature and ability to introduce specific genes into a cell…

T Cell Media: A Comprehensive Guide to Key Components

With the recent surge of cellular T cell-based immunotherapies, more and more researchers are taking an interest in these specialized immune cells. Whether you are brand new to the field or an experienced immunologist, learning to work with a new cell type can often feel overwhelming. In this guide, we will explain the ins and outs of culturing T cells, so you can spend less time searching for answers and more time making discoveries…

Cell Harvesting Technologies – Which is best for your process?

Cell harvesting is a critical step in connecting upstream monoclonal antibody production with downstream purification. Selecting the best cell harvesting technology based on the characteristics of the cell culture process can be challenging. This decision is made early in process development and requires a good understanding of both the current process and the advantages/disadvantages of the various cell harvesting technologies available…

Large Scale Adenovirus Production for Gene Therapy Manufacturing

Gene therapy is one of the most promising developments in medicine. It has the potential to treat many diseases by using a gene-modifying technology to repair, replace or correct genetic damage in the body. Last year was an important year for advancing gene therapy with two pioneering Chimeric Antigen Receptor T Cell (CAR T) cell therapies for cancer approved in the US: Novartis’ Kymriah^® and Kite/Gilead’s Yescarta^® and Spark’s Luxturna^® was approved to correct a retinal disease…

High Throughput Process Development in Biomanufacturing – Current Challenges and Benefits

Process development is and has always been a key component in successful scale-up of bioprocesses to commercial manufacture scale. At the Fourth High Throughput Process Development Conference in Toledo, Spain, several methods for high throughput process development were discussed for both upstream and downstream applications. Challenges and benefits were described along with ideas on where the industry can go from here. The proceedings were collected in a recently published report, “HTPD – High Throughput Process Development,” and include extended abstracts of talks presented at the conference. The report is very informative and I have summarized some key areas of focus in this article. We were also fortunate to be able to interview one of the conference chairs, Mats Gruvegard, GE Healthcare Life Sciences about what he saw as key takeaways from the conference and where he sees the future of high throughput process development heading…

Monolithic Chromatography Enables Process Intensification of Virus Purification

Monoliths are unique from other forms of chromatography media for several reasons. Monolith architecture consists of highly interconnected convective channels that are distributed throughout the entire bed. The large channels are easily available for purification even for large biomolecules. This unique architecture also creates a void-less space, thus significantly reducing shear and product loss. In monoliths, the mass transport is also exclusively convective and laminar which means that all solutes flow with the current regardless of size and require only a few seconds of residence time…

Rapid Characterization of Virus Like Particles Informs Vaccine and Gene Therapy Downstream Processes

When working with viral systems, either during manufacture of viral-based vaccines or gene therapies, having the right analytics is crucial. This is particularly true when optimizing downstream processes and in characterizing product lots for purity. Information on the amount of virus like particles present, particle structure and their biological impact not only informs the manufacturing process, but also final product purity and quality. While these answers are important they are not always easy to access. The following case study demonstrates the use of the MiniTEM as a convenient analytical, direct-measurement method to characterize virus like particles (VLPs) or virus preps. While this case study focuses on influenza vaccine, it is important to note that this is just one example and that the technology could be used in many viral-based vaccine or viral vector manufacturing scenarios…

A Guide for Selecting the Correct Filter Integrity Test Instrument

Selecting the right filter integrity test instrument is an important task. An informed, balanced choice will support the seamless integration of the instrument into critical processes and provide trouble-free operation for years to come. The right choice may be made more difficult by different departments, or user groups, who may assign a different value to the same feature. A rational approach is beneficial to ensure the selected instrument can fulfill all the selection criteria for all interested parties, with any compromises being limited to non-critical characteristics…

Downstream Manufacturing of Gene Therapy Vectors

The goal of downstream processing is to separate the viral vector from the various impurities produced during upstream processing and to get the virus into the appropriate state for formulation and administration to patients. Viral vector batches destined for clinical use must comply to increasing regulatory standards for impurities and contaminants as these can affect product safety and potency. In addition to providing purity, purification processes need to meet production scale, which can be quite large for clinical trials and commercial applications…

Headlines:

“A global coalition set up to fight disease epidemics is investing up to $8.4 million to develop a synthetic vaccine system that could be tailor-made to fight multiple pathogens such as flu, Ebola, Marburg and Rabies…”

“Orchard Therapeutics to Build Out Gene Therapy Manufacturing Site in Fremont, CA,” Genetic Engineering News

“Orchard Therapeutics said it will build out a gene therapy manufacturing facility in Fremont, CA, that will employ 100 people and increase its manufacturing capabilities to support plans for an expanded pipeline…”

“One of the biggest gambles in the history of the life sciences industry made it to Wall Street this evening when Moderna, a developer of messenger RNA drugs—an unproven technology with enormous potential—priced the largest biotech IPO ever…”

“Roche’s lung cancer combo treatment wins FDA approval,” Reuters

“Swiss drugmaker Roche Holding AG said on Thursday that its Tecentriq immunotherapy in combination with Avastin and chemotherapy won U.S. Food and Drug Administration approval as a first-line treatment for a type of lung cancer…”

“U.S.-EU Partnership on GMP Inspections Adds Five Countries,” FDA News

“The FDA has agreed to recognize drug and active pharmaceutical ingredient GMP inspections carried out by authorities in Belgium, Denmark, Finland, Latvia and Estonia, updating the 1998 mutual recognition agreement between the U.S. and the European Union…”

“CRISPR controls obesity in mice by amplifying rather than editing genes,” FierceBiotech

“The genes SIM1 and MC4R regulate food intake by signaling the feeling of being full, which is why mutations in those genes are associated with severe obesity. Even if just one of the two copies of those genes that a person inherits from both parents is bad, he or she will most likely feel hungry all the time…”