The cover story focuses on the first FDA-approved gene-transfer therapy, Novartis’s Kymriah, and other CAR T-cell products in development.
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.
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.
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.
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.
ISPE's new online course on implementing a Quality Manufacturing program considers FDA 2016 draft guidance
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 roles engineers play to drive continuous improvement. Part 2, slated for Pharmaceutical Engineering's...
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 precision, and it is expected to have profound implications for agriculture and the treatment...
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
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 called chimeric antigen receptor (CAR) T-cell therapies, which constitute a form of immunotherapy...
