This week’s headlines include: In a first, Gene Therapy Halts a Fatal Brain Disease, Most Accurate CRISPR Gene Editing Yet, RoosterBio and GenCure expand collaboration to provide end-to-end solutions for hMSC-based therapies, Novartis forges UC Berkeley pact, sees drug hopefuls in three years, Scripps Research-led study points to new flu drugs, Breast-cancer death rate drops almost 40 percent, saving 322,000 lives, study says, and FDA approves Novo Nordisk fast-acting insulin Fiasp.

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

Cool Tool – Efficient single cell isolation and proof of clonality in one step

Cell line development is a crucial part of biomanufacturing and sets the stage for future success by enabling the discovery of high yield and high quality clones. Within the cell line development workflow, one of the most critical steps is single cell isolation. The goal is to culture single cells in individual wells, which is necessary to ensure clonality. The FDA requires proof of clonality and companies must provide evidence that each cell line used in manufacturing has been generated from a single cell. This verification typically involves a combination of statistics and images to provide the required evidence…

Ask the Expert – Everything you ever wanted to ask about Corning® Matrigel® Matrix

Matrigel matrix is available is several formulations and can be used in a variety of different ways. With this flexibility comes the opportunity for expert advice and guidance to optimize the product selection and protocols most appropriate for particular cell types or applications. Corning recently published “The Ultimate Guide to Matrigel matrix” to share some of the best tips, tricks and expert advice. We encourage you to request your copy of the guide, available in both electronic and hard copy versions. And if you have more specific questions, we have two Corning Matrigel matrix experts hosting this Ask the Expert Session this week…

Transcriptome analysis reveals strategies for CHO cell culture media design and feed-spiking strategy to improve batch culture

When looking at batch vs. fed-batch culture, there are advantages and disadvantages to each, so considering the application is very important. Batch cultures are fairly simple, straightforward and take very little time to set up. However, batch culture typically doesn’t yield high mAb titers, due to nutrient depletion, by-product accumulation and short growth and production phases. So a batch approach may be good for lab-scale processes where product needs to be generated quickly and simply with little optimization. In contrast, with fed-batch culture, feeds are added to replenish nutrients, which increases cell concentrations, process time, and can yield much higher titers. However, fed batch culture also requires more time for optimization and resources to run. So this approach is more beneficial in situations where you would are developing long term processes or scaling up to large-scale production…

Automated integrity analysis of AAV and Adenovirus particles using MiniTEM

This study highlights the possibility of automated viral integrity measurements of Adeno-associated virus (AAV) and Adenovirus particles and underscores the importance of these measurements being performed in on-grid areas with appropriate stain quality. The analysis is performed using MiniTEM-enabled automated imaging, particle detection, classification and quantification. The analysis provides, a metric for determining the proportion of intact or broken particles in virus samples. In addition, as broken AAV particles may occasionally form undesired doublets or triplets, a metric for determining the proportion of such AAV particle formations is also demonstrated…

Cool Tool – ‘Jetting’ technology for manufacturing agarose beads with enhanced performance characteristics

The vast majority of chromatography resins designed for large-scale bioprocess chromatography separation are produced using traditional batch emulsification in conventional stirred-tank reactors. In these cases, the size of the beads formed in the reactor is a function of the shear force generated by the impeller. The faster the impeller speed, the smaller the beads are. As a result, there is a wide particle size distribution of the manufactured beads. Furthermore screening is required to remove coarse and fine beads, which detract from column performance. This screening is extremely time consuming particularly for smaller beads (less than 65 µm). The smaller the bead being produced the lower the achieved yield so realistically one cannot make beads financially viable less than 40 µm. It also adds high costs due to the additional time in the manufacturing facility with large volumes of waste from the fine and coarse beads. Even after this screening, the resin will still have a relatively wide size particle distribution…

Automated, single-use filtration to increase efficiency in upstream and downstream operations

In examining ways to improve overall bioprocess efficiency, filtration is a step that can sometimes be overlooked. However it is a key area to improve efficiency, as it is part of both upstream and downstream operations and consumes sizeable resources. The application of single-use technologies coupled with increased automation have successfully improved efficiency in other bioprocess operations, thus it is logical that filtration would also benefit from these technologies…

Bioburden Contamination in Downstream Bioprocesses – Potential entry points for contamination and innovative solutions

Bioburden contamination in biopharmaceutical manufacturing is a big concern. Contamination carries both tremendous cost and preventing it requires strict control of several possible entry points. The cost of bioburden contamination for a company can involve lost time, lost material, batch loss, possible facility closure and extensive QA/QC time to ensure proper cleaning and validation. In the worst case scenario, it can prevent supply of much needed medicine to patients and loss of commercial revenue…

Cool Tool – Achieve Integrated and Scalable Continuous Chromatography

Over the last decade, advances in the upstream processing of monoclonal antibodies (mAbs) has resulted in higher bioreactor titers. With increasing titers, the production bottleneck has shifted to downstream processing. Hence, the biopharmaceutical industry has reached a milestone where the need for higher throughput in downstream processing is leading to the adoption of more efficient multi-column continuous (MCC) counter-current chromatography systems which increase overall productivity while significantly reducing consumables costs…

Webinars:

Unmatched Viral Particle Analysis in Vaccine and Gene Therapy Development

October 18, 2017 at 10:00 EST

The number of Gene Therapy applications based on viral vectors are growing and small-scale manufacturing processes are increasingly requiring scaling up to commercial processes.Numerous challenges must be overcome in scaling-up to full commercial production Undesired outcomes may slip through if not monitored carefully during the process development.

This webinar will show how MiniTEM can reveal powerful data where traditional assays have limitations.

With the introduction of the MiniTEM™ system, transmission electron microscopy has become an accessible method for routine testing in bioprocessing. Through automated image acquisition, particle detection and classification MiniTEM can provide both high-resolution images and reliable quantitative data in a very short turnaround time.

Presented by Josefina Nilsson, Head of EM Services

Register to this webinar to learn more. – http://bit.ly/2i7TWYw

Events:

October

World Vaccine Congress
October 10 – October 12
CROWNE PLAZA BARCELONA, BARCELONA, Spain
Make sure you are at the forefront of the vaccines industry. No matter where your interest lies, at the 18th annual World Vaccine Congress we have content, networking and potential partners for you.

Speed to IND for Biologics
October 19 – October 20
Hyatt Centric Fisherman’s Wharf, 555 North Point Street
San Francisco, 94133 United States
With 32 expert presenters, 20 case studies/new data presentations and just two days out of the office, you won’t want to miss this first-of-its-kind event!

Global Pharmaceutical Regulatory Affairs Summit | 24-26 October 2017| Clarion Congress Hotel | Prague

Do you have a burning regulatory question? Come and get your queries answered with 4 parallel conference tracks on Regulatory Affairs in Emerging Markets, IDMP, Global eSubmissions and Biosimilars. We offer a new central European location, a focused agenda and more opportunities for you to collaborate and connect.

To learn more, please see – https://lifesciences.knect365.com/global-pharma-regulatory-affairs/

November

3rd Annual Cell & Gene Therapy Congress
November 6 – November 7
immarsat, 99 City Road
London, EC1Y 1AX United Kingdom

Oxford Global Conferences presents its 3rd Annual Cell & Gene Therapy Congress, with our co-located 6th Annual Cell Culture & Bioprocessing Congress and 4th Annual Stem Cell Congress and, 6 – 7 of November 2017, London, UK.

Cell & Gene Therapy: Development & Clinical Trials Cell Therapy Bioprocessing and Manufacturing Presentations will include cell & Gene Therapy development, updates in regulatory pathways, commercialisation, bioprocessing and manufacturing.

World Orphan Drug Congress Europe
November 13 – November 15
FAIRMONT REY JUAN CARLOS I, Av. Diagonal, 661-671
BARCELONA, 08028 Spain
The 8th annual World Orphan Drug Congress is the marketplace for orphan drug professionals looking at the complete value chain of orphan drug development, from clinical development and R&D to corporate development and market access.

Headlines:

“In a First, Gene Therapy Halts a Fatal Brain Disease,” The New York Times

“For the first time, doctors have used Gene Therapy to stave off a fatal degenerative brain disease, an achievement that some experts had thought impossible. The key to making the therapy work? One of medicine’s greatest villains: HIV…”

“Most Accurate CRISPR Gene Editing Yet,” The Scientist

“To minimize the off-target effects of CRISPR-based genome editing, researchers have designed a version of Cas9, the enzyme that cuts DNA, that avoids mistakes with unprecedented precision. Mutating one piece of the nuclease, called REC3, reduced off-target effects to below that of other high-fidelity Cas9 enzymes…”

“RoosterBio and GenCure expand collaboration to provide end-to-end solutions for hMSC-based therapies,” Globe Newswire

“Biotech organizations RoosterBio Inc. and GenCure announced today that they are expanding their existing collaboration by providing end-to-end solutions for hMSC (human bone marrow-derived mesenchymal stromal cell)-based therapeutic technologies. This biomanufacturing solution unites clinically relevant starting and ancillary materials with a scalable manufacturing process to deliver unprecedented adult cell manufacturing efficiencies. This expanded collaboration aims to shorten development timelines, simplify technology transfers, and deliver commercially viable cost structures, thereby to accelerate regenerative medicine removing years and millions of dollars from current commercial pathways…”

“Novartis forges UC Berkeley pact, sees drug hopefuls in three years,” Reuters

“Novartis’s NOVN.S research head expects an alliance with a University of California chemistry lab to produce drug candidates within three years as the Swiss company accelerates its hunt for new medicines. Novartis announced the Novartis-Berkeley Center for Proteomics and Chemistry Technologies on Thursday. It did not give financial details but said the multi-year deal targets “undruggable illnesses” like cancers that have eluded treatments…”

“Scripps Research-led study points to new flu drugs,” The San Diego Union-Tribune

“New and more powerful drugs to stop influenza may be possible, according to a study by scientists from The Scripps Research Institute and Janssen Research & Development, a unit of Johnson & Johnson. The study exploits a vulnerable site on the flu virus that existing drugs such as Tamiflu don’t target. Drug prototypes showed effectiveness in blocking flu infection, as shown in cell cultures…”

“Breast-cancer death rate drops almost 40 percent, saving 322,000 lives, study says,” The Washington Post

“Breast cancer death rates declined almost 40 percent between 1989 and 2015, averting 322,600 deaths, the American Cancer Society reported Tuesday. Breast cancer death rates increased by 0.4 percent per year from 1975 to 1989, according to the study. After that, mortality rates decreased rapidly, for a 39 percent drop overall through 2015.  The report, the latest to document a long-term reduction in breast-cancer mortality, attributed the declines to both improvements in treatments and to early detection by mammography…”

“FDA approves Novo Nordisk fast-acting insulin Fiasp,” Reuters

“The U.S. Food and Drug Administration on Friday approved Novo Nordisk’s fast-acting insulin to treat diabetes. The product, known as Fiasp, is designed to help diabetics control post-meal spikes in blood sugar. It is already approved in Canada and Europe…”