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.
Assurance of viral clearance can be difficult, however, during production of vectors such as adeno-associated viruses (AAV) used for the purpose of gene delivery. The need to define and deploy an appropriate and robust viral clearance strategy can be particularly challenging without an overarching strategy in place.
Protein viscosity is one of the major obstacles in preparing highly concentrated protein formulations suitable for subcutaneous injection. This whitepaper examines how combining an amino acid with a second viscosity-reducing excipient circumvents adverse effects on protein stability and improves viscosity-reducing capacity.
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.
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.
The manufacturing process for each viral vaccine is dictated by shape, size, nature, physico-chemical behavior, stability, and host specificity. This paper reviews steps in these manufacturing processes.