CELL & GENE ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS

• Identifying Heterogeneity Within Rare Cell Populations By Pairing Single-Cell RNA-Seq With Cell Sorting

  Studying biology at a single-cell level can provide crucial insights into how individual cells contribute to human biology and disease. Here we demonstrate that the resolution of single-cell RNA sequencing (RNA-Seq) can be greatly enhanced by adding a cell sorting step prior to RNA-Seq analysis.

• Registry And Natural History Studies: Vital, Contrasting Roles In Rare Disease Clinical Research

  Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success.

• Drug Substance Management With Single Use vs. Stainless Steel

  Fast freeze rates, safe storage and proper shipping of bulk drug substances are imperative. This case study compares the advantages and disadvantages of single-use systems and cryovessels.

• The Science Of Cell Therapy Thawing

  This guide summarizes the science of thawing following conventional, slow freezing methods. We address how cell thawing has historically developed into the techniques used today, along with the physical and biological implications of key metrics and components, such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.

• Switch To A Nitrogen-Free Cryopreservation System

  This study focuses on the running costs and carbon emissions associated with cryopreservation equipment, comparing a liquid nitrogen (LN₂)-free controlled-rate freezer with a typical liquid nitrogen-based system. The environmental implications of using liquid nitrogen for cell freezing are explored along with carbon emissions during the manufacture, transportation, and operation of LN₂-free controlled-rate freezers.

• Tumor-Agnostic Treatments: A Game Changer For Rare Oncology Trials

  As our understanding of the molecular characteristics of tumors has improved, there has been a tremendous leap forward — not only in targeted therapies but also in the development of tumor-or tissue-agnostic treatments. Now, with precision or personalized medicine, treatments can be targeted to a subgroup of patients rather than relying on a one-drug-fits-all model. In this blog post, we explore the changing rare oncology landscape and novel approaches to rare cancer trials.

• The Future Of MilliporeSigma’s Gene Therapy CDMO Through The Lens Of History

  On the road to becoming a global industry leader in cell and gene therapy, our viral vector manufacturing organization has undergone a major transformation over the last two decades. Marc Gaal, Director of Program Management at MilliporeSigma, details the history of the business as well as some of the milestones achieved since its inception.

• Developing A Continuous Large-Scale Perfusion Cell Culture Process

  The key benefits of perfusion processes are compact process design, flexibility, increased productivity, increased yield, and consistent product quality. 

• A Model To Increase Yield In mAb Cell Culture Perfusion At Large Scale

  As process intensification is adopted into large scale manufacturing, the responsibility of scale-down models to accurately represent the expanded operating space quickly follows. 

• Effective Downstream Purification: Clinical-Stage Retrovirus-Like Particles

  Purification of enveloped viruses and virus-like particles is challenging due to their size and complexity. This study demonstrates resin screening, process development, and scale-up purification approaches.