September / October 2017

September / October 2017 Issue Cover

Cover Story: A Breakthrough for Industry and Patients
On 30 August, the FDA approved Novartis’s Kymriah, the first gene therapy for the treatment of pediatric and young-adult patients with relapsed and refractory ALL. Kymriah belongs to a class of drugs called CAR T-cell therapies, a form of immunotherapy that redirects the body’s T-cells to attack tumors. This is the first FDA-approved gene-transfer therapy, but there are a number of other CAR T-cell products in development.

Feature: Patent Battles in the Age of CRISPR
CRISPR’s power lies in its relative simplicity and precision, and it is expected to have profound implications for agriculture and the treatment of disease. No surprise, then, that the groundbreaking technology is already beset with controversy ranging from ethical issues associated with the technology to a highly publicized and public US patent dispute.

Special Report: Setting the Course for Biopharma’s Future
While biopharmaceutical drugs have become standard therapy for multiple diseases, they require new manufacturing techniques both at the facility and throughout the supply network. When combined with changes in biopharmaceutical portfolios, these new technologies will create both challenges and opportunities for biopharmaceutical companies

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Features
The success of Novartis’s CTL019 in treating relapsed or refractory pediatric ALL, including FDA approval of the company’s biologics license application, makes it the first adoptive cell therapy to reach the market. While Novartis is also seeking...
Insights
Roughly 29,000 Americans die of Clostridium difficile infections every year. This gram-positive bacterium produces severe intestinal disease, with fatality rates ranging from 6% to 30%. Because the spores are hard to kill and can remain viable for years,...
Technical
The emergence of “big data” has allowed pharmaceutical organizations to harness the vast amount of information they generate. By collecting equipment, facility, and manufacturing data (process parameters, calibration, qualification, environmental...
Insights
A hero is someone who has given his or her life to something bigger than oneself. — "The Hero's Adventure," in ' The Power of Myth' , by Joseph Campbell with Bill Moyers, 1988
Features
On 30 August, the US Food and Drug Administration (FDA) approved Novartis's Kymriah (tisagenlecleucel, CTL019), the first gene therapy for the treatment of pediatric and young-adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia (ALL). Kymriah belongs to a class of drugs...
Special Reports
The portfolio sold by today’s global biopharmaceutical industry is fundamentally different than it was even a decade ago. This shift is a reflection of today’s global market, which features greater competition, more treatments for orphan diseases, an...
InTouch
The ISPE Baseline® Guide: Risk-Based Manufacture of Pharmaceutical Products (Risk-MaPP) (second edition), provides a scientific risk-based approach based on ICH Q9: “Quality Risk Management,” for managing the risk of cross-contamination within shared...
InTouch
Since the Turkish pharmaceutical industry began applying good manufacturing practices and other international standards in 1984, its performance has been strong. Today, Turkey is home to 300 pharmaceutical companies and 31,000 employees, which produce...
InTouch
Antonio Moreira, PhD, is unequivocal about ISPE’s need to encourage members and colleagues around the world. “They have their challenges regarding travel and sometimes political unrest, so anything we can do that demonstrates ISPE’s commitment to support...
Features
The CRISPR/Cas9 system (CRISPR) developed for gene editing only a few short years ago* has already revolutionized genomic engineering, and clinical trials for targeted cancer therapies have recently commenced in China and the United States. CRISPR's power lies in its relative simplicity and...
InTouch
This is an important event for quality professionals," said Marianne Bock, ISPE’s director of continuing education, describing the fifth annual ISPE/FDA/PQRI Quality Manufacturing Conference, held 5–7 June in Arlington, Virginia, United States. "This...
Insights
Many spend their summer relaxing, perhaps traveling, but generally trying to “put their feet up.” Others change the course of health care as we know and practice it. Gilead’s 28 August purchase of Kite Pharma, one of the companies developing chimeric...
InTouch
I was first asked to be a workshop leader for the Quality Manufacturing Conference this past June, I accepted thinking my workshop assignment would be directly related to my professional field of architecture. Instead, I was completely out of my comfort...
InTouch
ISPE celebrated the thirteenth annual Facility of the Year Awards (FOYA) banquet on Tuesday, 6 June, with a lively group of participants from the ISPE/FDA/PQRI Quality Manufacturing Conference and beaming category award winners in attendance. Guests...
InTouch
Do you want to connect with your colleagues around the globe? Are you seeking information on current trends, hot topics, or how to solve a problem? Then look to the ISPE Communities of Practice! CoPs are virtual libraries, a group of online communities...