September / October 2017

September / October 2017 Issue Cover

 Breakthrough for Industry and Patients

Cover Story: 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. This is the first FDA-approved gene-transfer therapy, but there are a number of other CAR T-cell products in development.

Patent Battles in the Age of CRISPR

Feature: 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.

Setting the Course for Biopharma’s Future

Special Report: While biopharmaceutical drugs have become standard therapy for multiple diseases, they require new manufacturing techniques both at the facility and throughout the supply network.

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...
Special Reports
China’s biological pipeline may take the lead China and India have demonstrated their capability in good manufacturing practice (GMP) manufacture of small-molecule drugs for decades. But...
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...
Special Reports
As the industry evolves in its quest to increase quality, productivity, efficiency, and safety, engineers must be equipped with skills that enable them to meet new and challenging demands. This first in a two-part series focused on biopharmaceutical manufacturing explores a few of the exciting...
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...
Special Reports
This article was developed by members of the ISPE Biotechnology Community of Practice. The views and opinions are those of the authors and do not necessarily reflect the official policy or position of Hargrove Life Sciences, M+W, Novartis, Sandoz, Takeda, or any of their officers.