This week’s headlines include: Cancer Patient Climbs Mountains, FDA to fast track Mesoblast’s Cell Therapy for heart failure, FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements, Johnson & Johnson pens Alzheimer’s Gene Therapy pact, South Korea’s Samsung Bioepis says FDA to review Herceptin copy, and Novartis breast cancer therapy gets FDA breakthrough designation.

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

Novel technology enables encapsulated cell therapeutics without triggering fibrosis

In this podcast and accompanying article, we interviewed Dr. Paul Wotton, CEO, about Sigilon Therapeutics’ discovery platform. The platform combines cell engineering and biocompatible Afibromer^™ technology to enable cell therapeutics that do not trigger fibrosis…

Cool Tool – Xeno-free media supplement for Cell Therapy Manufacturing

Cell-based therapy has demonstrated significant promise in the potential to treat some of the most challenging diseases. The recent FDA approvals of Novartis’ Kymriah and Kite Pharma’s Yescarta demonstrate the tremendous benefits this industry has to offer. As more and more cell therapies progress towards commercialization, safe and efficient manufacturing becomes critically important. Because the cell is the therapy, materials and methods used to culture the cells are a key focus of regulators. It is easy to become inundated by the plethora of reagents available on the market and risk mitigation strategies are becoming differentiators. Several companies have looked to serum-free culture in an attempt to remove the variability associated with using animal source sera. However, the ex vivo manipulation necessary for Cell Therapy manufacturing creates a more challenging system for the cells to survive in and thus they have a tendency to struggle in serum free conditions. If the cells aren’t growing well, then manufacturing can’t meet the efficiency or timelines required to create a viable product…

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…

Protein A Chromatography – A look at where we have been and where we are going

In this podcast and accompanying article, we interviewed Jonathan Royce,  Business Leader, Chromatography Resins, GE Healthcare Life Sciences, about the evolution of Protein A including the latest developments in Protein A chromatography resins. We also discussed what the future holds for this purification mainstay and how it can continue to address the changing needs of biopharma…

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…

Podcasts:

Traveling over the holidays and need something to listen to? Check out our podcast channel. We have 15 great podcasts covering drug discovery, stem cell culture, upstream and downstream biomanufacturing and more!

Click below to download through iTunes or Google play:

Headlines:

“When the Lung Cancer Patient Climbs Mountains,” The New York Times

“On Oct. 15 at 8 a.m., Andy Lindsay stood atop 21,247-foot Mera Peak in Nepal, a wildly improbable place for him to be both athletically and medically. Andy, a veteran climber and a friend of mine, had been living with Stage IV lung cancer for three years. “To live one year was statistically unlikely, and two years looked like a miracle,” he said. He was able to make the climb thanks to the success of a cutting-edge Gene Therapy clinical trial. It targeted his specific lung cancer mutation, shutting off the fuel to his tumor’s growth and shrinking the tumor. He wasn’t cured, but his scans were strikingly improved and he was almost symptom-free…”

“FDA to fast track Mesoblast’s Cell Therapy for heart failure,” Reuters

“Australian drug developer Mesoblast Ltd said on Thursday the U.S. Food and Drug Administration would fast track the review of its Cell Therapy treatment for heart failure patients under a new designation rolled out last December. Mesoblast’s mesenchymal precursor cell (MPC) therapy, aiming to mitigate advanced heart failure, received the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation, the company said…”

“FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements,” Regulatory Focus

“The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions…”