Chemistry Manufacturing and Controls (CMC) for gene therapies is one of the biggest obstacles when moving towards regulatory approval and presents a significant risk to the success of new gene therapy drug candidates today. Here we review a framework for a QbD assessment for AAV products within the Chemistry Manufacturing and Controls (CMC) documentation.

Local delivery of biotherapeutics to the lung holds great promise for treatment of lung diseases, but development of physically stable, biologically active dry powder formulations of large molecules for inhalation has remained a challenge.

Clone selection is a significant upstream bottleneck slowing bench-to-bedside development progress for new mAbbased therapeutics. With mAb-based product development addressing diseases of such massive financial and societal implications, researchers using analytical power tools can reach their goals faster and shorten the bench-to-bedside development path, benefitting both patients and the bottom line.

Cell line development involves multiple processes. Large numbers of clones are screened and selected on the basis of productivity and stability. An established platform for rapid titer of antibody clones can enable quick selection of high-producing clones.

Dr. Gene Lee, Vice President of Technical Development at AltruBio, sat down to discuss getting the developability question right and why it’s important to understand your end game as early as discovery.

Geoff MacKay, president and CEO of AVROBIO, talks with The Business of Biotech podcast about the scale up obstacles facing the cell and gene therapy space and how AVRO is working to tackle them.

While current approaches can manage the existing volume of cell therapy shipments, scaling these treatments up and out will increase pressure on cryogenic logistics networks. Here, we consider the challenges and risks associated with the cell therapy cryogenic supply chain and provide considerations for mitigation strategies.

A leading organization in small molecule therapies was facing a challenge in ensuring consistent CPV initiatives across their different manufacturing sites was maintaining a uniform standard of data in their manufacturing analytics. A software solution that was purpose-built for biopharmaceutical manufacturing provided a strong foundation to streamline the validation of manufacturing analytics for CPV and reporting.

To ensure that a commercial biomanufacturing process is in a state of control, life science companies must create and successfully execute initiatives to meet continued process verification (CPV) and other monitoring guidelines. Learn how to navigate the major steps of implementing a global monitoring plan for continued process verification.

bluebird bio's Scott Cleve recently sat down with The Business of Biotech to talk about shifting regulatory standards and how pharmas can adapt how they engage with regulators.