The Dish’s Weekly Biotechnology News Wrap Up – July 21, 2017
This week’s headlines include: Vertex Touts Positive Triple-Drug Data For Cystic Fibrosis Patients, Emergent Buys Sanofi Pasteur’s Smallpox Vaccine for Up-to-$125M, Puma to price new cancer med Nerlynx at a higher-than-expected $10K per month: report, Emergent BioSolutions to acquire anthrax treatment, move manufacturing to Baltimore, Gottlieb: FDA to Issue new Policies to Streamline Generic Reviews in 2017, and Scientists plan to trick Zika-carrying mosquitoes into breeding themselves out of existence.
In Case You Missed It, Recent Articles on Cell Culture Dish and Downstream Column:
In-line Viral Load Measurement using Smart Cell Culture Monitoring
Smart in-line cell culture monitoring as an efficient way to measure viral load in real-time. This kind of real-time measurement is only possible with the iLine F. The iLineF is an innovative microscopy technology that instead of taking a 2D image of a microscopic object, takes a hologram of a microscopic volume. Then for each microscopic object within the culture volume, it can compute a holographic fingerprint. This fingerprint can then be used to analyze, identify, count and assess viability of cells in culture…
Characterization and analysis of subvisible particles of biological origin can be challenging or give insufficient information. In this study Adeno associated virus (AAV) particles contaminated by host cell proteasomes are analysed with direct versus indirect methods performed in a standard laboratory setting to reveal the difference in performance and quality of the information obtained. The focus is on the ability to detect the AAV particles of interest and distinguish them from the contaminating proteasomes…
Hematopoietic stem and progenitor cells (HSPCs) hold tremendous therapeutic potential for use in cell therapies to treat blood cancers and other blood diseases including sickle cell anemia and immune related disorders. HSPCs most frequently are sourced from donor blood or bone marrow. These cells are rare with only 1 in 100,000 found in peripheral blood cells and 1 in 10,000 found in bone marrow. In order to achieve the dosage required for most cell therapies (100’s of millions), cells must be successfully expanded to high numbers in culture. However during expansion, cells must also maintain the progenitor cell properties including the potential to differentiate and mature into the various hematopoietic lineage cells required for therapeutics…
First In-Human Allogeneic Clinical Trial Commences with iPSC-derived Mesenchymal Stem Cells
It is widely accepted that stem cells can be divided broadly into embryonic and non-embryonic stem cells. Embryonic stem cells (ESCs) are derived from the inner cell mass of blastocysts and are pluripotent, meaning they can differentiate into cells of all three germ layers: ectoderm (outer layer), mesoderm (middle layer), and endoderm (inner layer). Conversely, non-embryonic stem cells are found in the extra-embryonic tissues (placenta, umbilical cord blood and amniotic fluid) and in all adult tissues, (i.e. bone marrow, fat, kidney, etc). Human mesenchymal stem cells (hMSCs) are an example of non-embryonic stem cells and were first isolated in the bone marrow and characterized by Friedenstein and his colleagues in 1974 (Amorin, 2014). hMSCs, also called mesenchymal stromal cells, are a subset of non-hematopoietic adult stem cells that originate from the mesoderm (Kim et al, 2013). They are considered to be multipotent; able to self-renew and generate progeny of several distinct cell types…
Fine Tuning Viral Clearance Approaches with a Total Viral Challenge Strategy
In this mini-webinar, Michael Burnham, M.S., Senior Principal Scientist, Process Development and Commercialization, WuXi AppTec, presents a viral clearance strategy that focuses on spiking load or starting material based on total viral load instead of percent spike model…
Continuous bioprocessing – moving from theory to reality
Continuous manufacturing has been established in several processing industries for many years, providing many benefits over batch manufacturing. The feasibility of continuous processing has now been shown for monoclonal antibodies (mAb) at both the process development (PD) and the production scales by early adopters…
Subvisible Particle Characterization: Why Simply Counting Shadows Leaves You in the Dark
Significant advances in analytical technology over the past few years have improved the quantification and characterization capabilities for subvisible ( 1 – 100 µm) and submicron particles (≤1 µm). As the technology continues to improve so do the expectations of regulatory agencies for sponsors to characterize particles in these size ranges. However, multiple orthogonal methods are required to span the entire range and accurately characterize the particle profile. Each instrument has its own limitations based on detection method and properties of therapeutic protein products that must be well understood to generate high quality data. KBI Biopharma has extensive experience with particle detection methods, as well as, in-depth particle data analysis. KBI’s Particle Characterization Core team can help choose appropriate orthogonal particle to combine in order to accurately quantify, characterize and identify particles in specific therapeutic protein products for all size ranges based on clients’ needs
Multiple buffers in a wide range of formulations are required to produce a single biopharmaceutical. Because of the large volumes required, buffer preparation can easily become a bottleneck in production. Traditionally, buffers are prepared manually in the volume needed according to specific recipes. Due to the large quantities used, buffer management requires careful planning and considerable floor space is required for the preparation and storage of such large buffer quantities. In addition to high labor and facility space cost, there is a risk of human error and variability associated with such a time-intensive manual activity. Buffer variability can affect both quantity and quality of the final product…
Headlines:
“Vertex Touts Positive Triple-Drug Data For Cystic Fibrosis Patients,” Xconomy
“The Boston firm announced Tuesday that studies of three experimental drug combinations all reported notable improvement in patients who have a different underlying genetic pattern than patients already eligible for current commercial products. If all plays out well, up to 90 percent of people with CF could have a drug to treat their disease in a few years…”
“Emergent Buys Sanofi Pasteur’s Smallpox Vaccine for Up-to-$125M,” Genetic Engineering News
“Emergent BioSolutions has agreed to acquire the ACAM2000® [Smallpox (Vaccinia) Vaccine, Live] business of Sanofi Pasteur for up to $125 million cash, in a deal the buyer said would reinforce its strategic focus on treating public health threats…”
“Puma to price new cancer med Nerlynx at a higher-than-expected $10K per month: report,” FiercePharma
“Shares of Puma Biotechnology have tripled since the start of the year to more than $95, as the Los Angeles company gets set to launch its debut drug, Nerlynx (neratinib) for HER2-positive breast cancer. Now analysts are predicting premium pricing for the new drug that they believe will drive shares even higher…”
“Emergent BioSolutions to acquire anthrax treatment, move manufacturing to Baltimore,” Baltimore Sun
“Emergent BioSolutions, a Gaithersburg-based drug maker with facilities in Baltimore, has acquired a drug used to treat anthrax from GlaxoSmithKline in a deal worth up to $96 million…”
“Gottlieb: FDA to Issue new Policies to Streamline Generic Reviews in 2017,” Regulatory Focus
“By the end of 2017, the US Food and Drug Administration (FDA) will issue two new documents to improve the review process for generic drugs, according to FDA Commissioner Scott Gottlieb...”
“Scientists plan to trick Zika-carrying mosquitoes into breeding themselves out of existence,” The Washington Post
“This summer, a Silicon Valley tech company will have millions of machine-raised, bacteria-infected mosquitoes packed into windowless white vans, driven inland and released into the wild — or, at least, the streets of Fresno, Calif. And, yes, Fresno County officials are encouraging this. It’s all part of the “Debug Fresno” project, which aims to cut down on the number of Aedes aegypti mosquitoes, an unwelcome invasive species that arrived in California’s Central Valley in 2013. In addition to being potential carriers of the Zika, dengue fever and chikungunya viruses, the Aedes aegypti also adapted rapidly to the area’s residential neighborhoods, to the chagrin of residents and officials alike…”