This week’s headlines include: Trial results of Zika vaccine Sanofi dropped show promise, Out-of-pocket health spending in 2016 increased at the fastest rate in a decade, FDA proposes drug development guidance for rare pediatric diseases, 1-year data set Spark/Pfizer on track with hemophilia B Gene Therapy, Shire, Rani to develop oral factor VIII therapy, FDA approves heart protection claims for Amgen cholesterol drug, and Mustang Bio Launches CRISPR/Cas9 CAR-T Collaborations with Harvard, BIDMC.

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

Dissolvable microcarriers – Efficient and streamlined stem cell harvest for Cell Therapy biomanufacturing

We recently finished our Ask the Expert discussion, “Enabling Stem Cell Therapy Biomanufacturing using Dissolvable Microcarriers.” During this Ask the Expert session, we covered topics related to the large scale culture of stem cells for Cell Therapy and the use of dissolvable microcarriers as an enabling technology. Topics included general questions about the use of microcarriers in Cell Therapy manufacturing, for example, microcarrier optimization, cell aggregation when using microcarriers, and recommended scale for switching to microcarriers. In addition, we had several questions related to the use of dissolvable microcarriers that included, compatible cell types, cell attachement, cell harvest, media, bioreactor compatibility, and durability…

Development of an Improved Poloxamer 188 – Optimized for Cell Culture Performance

Poloxamer 188 is a surface-active non-ionic polymer used in cell culture media as shear protectant. With time it became a standard ingredient in cell culture media for commercial production processes. It was demonstrated to increase the robustness of mammalian cells to shear from sparging, which probably is the strongest contributor to the hydrodynamic stress in bioreactors…

Video – Upstream Bioprocessing Cell Culture Overview

An overview of upstream bioprocessing steps beginning with an explanation of molecular biology and cell line engineering, then continuing with creation of a working cell bank, cell expansion, bioreactor production and clarification steps…

Organoid Cell Culture – Frequently Asked Questions

We recently finished our Ask the Expert discussion, “Going tiny is the next BIG thing: Tools and Techniques for Organoid Cultures”. During this Ask the Expert session, we covered topics related to the culture of organoids including, media requirements, co-culture with other cells, surface requirements, culture vessels, nutrient delivery and gas exchange, harvesting organoids, and key points on the cell culture process timeline. In addition, we also had questions about making organoid cell culture more high throughput to meet the needs of drug screening, the difference between spheroids and organoids, and best practices for immunostaining of organoids…

Downstream Bioprocessing Cost Modeling – Looking at Integrated Continuous, Single-use and Stainless Steel Platforms

Process economics are frequently discussed with respect to continuous biomanufacturing implementation. More specifically, are the potential cost benefits worth making a manufacturing change and in which situations are the benefits greatest? At Biotech Week Boston in September, there was a very interesting talk titled “Cost modeling of the downstream bioprocessing design space,” presented by Mark Schofield, Ph.D., Senior R&D Manager, Pall Life Sciences. In the talk, Dr. Schofield shows data related to cost modeling the downstream bioprocess design space. He also describes some of the challenges facing biomanufacturing including cost pressure, competition, and the rise of biosimilars, and how implementing integrated continuous operations can address several of these challenges…

Resolving large scale buffer management challenges

In this podcast and accompanying article, we interviewed Joakim Lundvist, Modality Manager, BioProcess™ Hardware, GE Healthcare about large-scale buffer management challenges. Buffer preparation is known to be one of the most resource-intensive activities in biomanufacturing as large volumes of buffers and process liquids are often required. So how can this be done in a more efficient way? How can more capacity be added to buffer preparation without adding major capital investment? Are there ways to reduce the manufacturing footprint and time spent on buffers?…

Enabling Antibody-drug conjugate manufacturing using single-use systems in downstream – Extractables study demonstrates a good fit

Antibody drug conjugates offer tremendous therapeutic potential and the market for ADCs is expected to expand rapidly. However, antibody-drug conjugate manufacturing presents both technological and logistical challenges…

2017 Biotech Week Boston – Downstream Coverage Highlights

Last month, Boston Biotech Week featured interesting content with a wide range of issues, from cell culture techniques to commercialization and everything in between covered. Talks focused on improving the manufacturing process for biopharmaceuticals and cell therapies, enabled industry networking opportunities, and provided the chance to see the latest products and technologies…

Webinars:

Improving NK Cell-based Cancer Immunotherapy via mRNA Electroporation

December 13, 2017 – 11:00 am EST

Natural Killer (NK) cells are cytotoxic lymphocytes that can kill tumor-transformed and infected cells. In contrast to T cells, genetic engineering of NK cells using viral vectors has only recently started to show sufficient efficacy. Currently, viral approaches are associated with high costs and excessive regulatory work-load if used in the clinic. Genetic engineering of NK cells via mRNA electroporation has instead shown to be more efficient than viral transduction and is likely associated with less regulatory work-load for clinical realization. This webinar will focus on the current developments of mRNA electroporation of NK cells, including its efficacy and impact on cellular viability, proliferation, receptor expression and baseline cytotoxicity. Examples will be discussed for how mRNA electroporation can be used to modulate multiple properties of NK cells and thereby potentially further improve the outcome of cancer patients treated with NK cell-based immunotherapies in the future. Register Now!

Headlines:

“Trial results of Zika vaccine Sanofi dropped show promise,” Reuters

“A Zika vaccine Sanofi SA dropped in September under political pressure over pricing produced strong responses in more than 90 percent of those taking part in an early-stage clinical trial, U.S. researchers reported on Monday…”

“Out-of-pocket health spending in 2016 increased at the fastest rate in a decade,” Washington Post

“U.S. health care spending increased to $3.3 trillion in 2016, with out-of-pocket health care costs borne directly by consumers rising 3.9 percent — the fastest rate of growth since 2007…”

“FDA proposes drug development guidance for rare pediatric diseases,” Reuters

“The U.S. Food and Drug Administration issued a draft guidance on Wednesday to simplify the procedure of developing drugs for rare pediatric disorders, such as Gaucher’s disease, by eliminating the need for certain trials and minimizing patient enrollment…”

“1-year data set Spark/Pfizer on track with hemophilia B Gene Therapy,” Fierce Biotech

“Patients with hemophilia B have seen their symptoms controlled for up to a year with a single dose of a Gene Therapy developed by Spark Therapeutics, almost entirely eliminating the need for treatment with clotting factors…”

“Shire, Rani to develop oral factor VIII therapy,” PharmaTimes

“Shire and Rani Therapeutics have signed a deal to work on developing an orally-delivered therapy for patients with hemophilia A…”

“FDA approves heart protection claims for Amgen cholesterol drug,” Reuters

“Amgen Inc can now promote the ability of its potent but expensive cholesterol drug to reduce the risk of heart attacks and strokes, after U.S. health regulators approved adding those benefits to the medicine’s prescribing label, the company said on Friday…”

“Mustang Bio Launches CRISPR/Cas9 CAR-T Collaborations with Harvard, BIDMC,” Genetic Engineering News

“Mustang Bio said today it plans to develop CRISPR/Cas9-enhanced chimeric antigen receptor engineered T-cell (CAR-T) therapies for cancer through a license from Harvard University and a research collaboration agreement with Beth Israel Deaconess Medical Center (BIDMC)…”