Cynthia A. Challener
Scientific Content Director
That’s Nice, LLC

Abstract

Initial clinical successes in a wide variety of indications and commercial launch of three cell and gene therapy products have driven significant investment in these next-generation technologies. Both large bio/pharma and start-up companies are eager to advance beyond early clinical trials to Phase III and commercial. As part of the BioPharm Insights event at CPhI Worldwide 2016, representatives from different contract development and manufacturing organizations (CDMOs) discussed the numerous challenges facing producers of these and other state-of-the-art medicines as they scale up manufacturing processes. The panel also discussed the role that CDMOs will play in facilitating the advancement of these technologies. A summary of the discussion is presented below.

REAL PROMISE
Many of the most promising next-generation therapies under development today are based on new biologic drug entities that require the development of new technologies for their manufacture, characterization, testing and distribution at commercial scale.

These new treatments have for the most part only been produced on very small scales for early clinical trials, and orphan drug applications, either in-house by biopharma company process development labs or academic groups that provide small-scale contract manufacturing services.
The methods used to produce, analyze and handle such small quantities are in general not sufficient for meeting the demands of the full clinical population, nor economically viable or practical at commercial scale.

MANAGING STRUCTURE AND FUNCTION IN PROTEIN-BASED NEXT-GEN BIOLOGICS
Antibody fragments, multi-specific antibodies and nanobodies each represent different challenges at various stages of development, according to Gustavo Mahler, CEO of CMC Biologics. “With bispecific antibodies, the challenge starts with cell line development, where it is necessary to ensure th ...