This week’s headlines include: Novartis’s Kymriah wins speedy reviews in U.S., Europe, Stanford Spinout Partners with Merck KGaA, Genentech in Cancer Studies of Hu5F9-G4, After a Debacle, How California Became a Role Model on Measles, Drugmakers see a pricing blueprint in an $850,000 Gene Therapy, Researchers build immune systems for kids with ‘Bubble Boy’ disease, Gilead/Kite team up with Pfizer for CAR-T-mAb combination, and Transparency: FDA to Release Portions of Redacted Clinical Study Reports.

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

The Next Level of Quality for Cell Therapy Media – Beyond Xeno-Free

Since the advent of in vitro biology, mammalian cell culture and cell culture-based assays have served as an indispensable tool in the elucidation of biological processes. This article will discuss the current landscape of FBS and human serum use and their limitations, as well as introduce a powerful alternative…

Theranostics: an old approach with new interest

Precision medicine has been a growing trend in healthcare for years. Doctors and researchers are leveraging computer power, modern analytics, cloud technology and patient information from genomics to demographics to help make more individualized and accurate treatment plans. What if there were a more direct link between a diagnostic test and the potential therapies available for a patient? Well that is the aim of theranostics, a field of personalized and precision medicine that links targeted therapies to specific diagnostic tests. Theranostics represents one approach for transitioning from conventional medicine to a more contemporary patient-specific analysis and therapy (1). While having evolved into a number of particular approaches, a theme currently in vogue is the use of either micro- or nano-particles in a marriage of diagnostic imaging and therapeutic activities. This results in the use of a single nano-particle agent to be employed in screening, diagnosis and treatment delivery as well as in prognosis or response monitoring…

Ask the Expert – Implementation of Continuous Manufacturing Strategies for Protein Therapeutics

There continues to be growing interest in continuous processing of biological therapeutics with the primary driver being reduction of drug costs through more efficient manufacturing processes. Together, drug developers, contract manufacturing organizations and process equipment suppliers continue to make significant advancements towards the goal of making continuous biologics manufacturing a reality from the initiation of cell culture to the production of bulk drug substance. Compared to traditional batch technologies, continuous bioprocessing can offer more flexibility and in some cases improve product quality in addition to lowering the costs of production. Continuous cell culture technologies can be used in the manufacturing of both stable and unstable biological molecules. The US FDA has been supportive of the implementation of continuous manufacturing using a science- and risk-based approach…

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…

The Top 25 Downstream Column Blogs of 2017

I have compiled a list of our most popular 25 blogs, podcasts, and webinars for 2017 listed in alphabetical order…

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?…

Webinars:

The Process Economics of Continuous Downstream Bioprocessing

New Webinar Available On Demand!

The cost of goods of an Integrated Continuous Bioprocessing (ICB) platform for the manufacturing of a mAb from fed-batch cell harvest has been evaluated using BioSolve Process, a modeling software from BioPharm Services. The costs associated with the ICB platform were compared to those of stainless steel and single-use batch processes. These downstream processes were evaluated across sets of clinical and commercial production scenarios. For both sets of scenarios, 27 different cases were modeled by varying 3 factors: bioreactor titer, volume, and number of batches per year. By taking this approach, the cost-benefits of the ICB platform could be seen across a wide range of manufacturing scales.

In this webinar, participants will learn:

• The findings of the full-factorial modeling exercise.
• The cost-benefits of continuous bioprocessing at many scales of manufacturing.

Podcasts:

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:

“Novartis’s Kymriah wins speedy reviews in U.S., Europe,” Reuters

“Novartis has been granted fast-track reviews of its Cell Therapy Kymriah for blood cancers in the United States and Europe, the Swiss drugmaker said on Wednesday, as it seeks to expand the pool of patients eligible for the costly treatment…”

“Stanford Spinout Partners with Merck KGaA, Genentech in Cancer Studies of Hu5F9-G4,” Genetic Engineering News

“Forty Seven Inc., a developer of immuno-oncology treatments spun out of Stanford University, has launched a pair of collaborations with pharma giants Merck KGaA and Genentech, a member of the Roche Group, to develop its lead candidate as part of combination therapies for a trio of cancer indications…”

“After a Debacle, How California Became a Role Model on Measles,” The New York Times

“In December 2014 something unusual happened at Disneyland. People came to visit Mickey Mouse, and some of them left with measles. At least 159 people contracted the disease during an outbreak lasting several months. This is more than the typical number in a whole year in the United States…”

“Drugmakers see a pricing blueprint in an $850,000 Gene Therapy,” Reuters

“Global drugmakers are looking to a tiny biotech’s $850,000 therapy for a rare type of blindness as a model for getting paid for highly expensive – and effective – new medicines…”

“Researchers build immune systems for kids with ‘Bubble Boy’ disease,” USA Today

“A new type of Gene Therapy pioneered at St. Jude Children’s Research Hospital appears to build fully functional immune systems in babies born with the so-called “Bubble Boy” disease, offering hope of a permanent cure for kids who otherwise might die before their second birthdays…”

“Gilead/Kite team up with Pfizer for CAR-T-mAb combination,” FierceBiotech

“Gilead and its shiny new $12 billion biotech buy Kite Pharma have teamed up with Big Pharma Pfizer to work on a Yescarta-utomilumab combination test in certain blood cancers. Yescarta (axicabtagene ciloleucel), approved by the FDA last year in the first wave of new CAR-T cell therapies, will be teamed up with Pfizer’s experimental humanized 4-1BB agonist utomilumab in patients with refractory large B-cell lymphoma…”

“Transparency: FDA to Release Portions of Redacted Clinical Study Reports,” Regulatory Focus

“As part of a push to be more transparent about its decision making and enhance access to data for researchers, the US Food and Drug Administration (FDA) on Tuesday announced it will launch a pilot project to publicly release portions of clinical trial-related summaries from pivotal trials after a drug is approved…”