H - Hybrid

With the significant progress in the development of individualized medicines, several issues with respect to their routine quality testing have emerged…

Potency testing is a critical part of the assessment and release of cell therapy products. These functional assays provide quantitative assessments of the biological activity of a cell or gene therapy product that are associated with that product’s in vivo mechanism(s) of action (MOA)…

Many cell and gene therapy products use special delivery devices. Generally, there are two approaches for development and commercialization. 1: specify a device brand in the label to be used with the cell or gene therapy products…

Gene editing holds the promise to revolutionize medicine due to its potential to correct or disrupt target DNA sequences with a putative long-lasting effect…

The further advance of techniques for genetic modification also necessitates development of novel and more comprehensive genetic characterization tools to identify intended and unintended genetic changes in (subpopulations of) genetically modified cells…

Decentralized manufacturing involves the use of manufacturing units that can be deployed to multiple locations and in cases, to sites close to the patients…

Cell and Gene Therapy programs are progressing from early academic settings to commercial manufacturing at increasingly faster pace and in greater numbers. These therapies are complex, difficult to manufacture in large quantities, and involve many bespoke technologies to make, test and release…

Comparability assessments for cell-based therapies are challenging due to complex manufacturing processes, inherent variability of cell starting materials, and evolving understanding of product quality attributes and analytical technologies…

Viral vector-based gene therapy products are the fastest growing segment of the gene therapy field, with adeno-associated virus (AAV)-based vectors dominating the landscape…

With the recent approval of several Adeno-Associated Virus (AAV)-based gene therapies, the application of AAV gene therapy approaches to the treatment of human disease is expanding…

Bringing treatment to those afflicted with an ultra-rare disease is complex. These products are encumbered with challenges ranging from high development costs with a limited ROI, to issues with executing and interpreting clinical trials with unusually small patient populations…

Allogeneic cell therapies have a potential to revolutionize life-saving Cell and Gene therapy (CGT) field by removing the bottlenecks created by long scheduling delays, lengthy manufacturing times and potential product failures of autologous cell therapies…

Several cell and gene therapy products have received marketing approval from health authorities and, with that, a more complete picture is now emerging about the regulatory CMC requirements to commercialize these products…

Analytics for Process-related Impurities in Viral Vector Manufacturing 

Raw material selection decisions early in the development of ATMPs are crucial and can impact the final product quality and ultimately patient safety…

Ensuring the Virus Safety Profile of Your Product - Expectations and Recommendations From the Latest Update to ICH Q5A

Gene Therapy Drug Development for Ultra-Rare Disease: Challenges & Opportunities

Comparability assessments are necessary for life cycle management of all biological products, including cell and gene therapies (CGT), and are conducted to ensure that manufacturing changes do not adversely impact product quality, safety, or efficacy…