Cell And Gene Articles, App Notes, Case Studies, & White Papers

1. Alternative To Cryobags For Large Volume Cryopreservation 6/6/2019

   This study demonstrates an optimized cooling protocol for cryopreserving large-volume cryovials in a controlled-rate freezer, avoiding the need for liquid nitrogen.

2. Identifying Heterogeneity Within Rare Cell Populations By Pairing Single-Cell RNA-Seq With Cell Sorting 6/7/2019

   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.

3. Quality System Overview For GMP Manufacturing 11/26/2019

   When you think about cell and gene therapies (CGTs), what most often comes to mind is the early stage scientific innovation responsible for finding new ways to treat diseases. Unfortunately, what is sometimes overlooked or left to the last minute is the less-than-glamourous, but still critical, manufacturing steps that ensure a new, potentially lifesaving treatment reaches patients in a safe and reliable form. Following is an overview of the mandatory quality systems and functions that are required within a standard GMP facility to comply with ICH Guidelines including Q7 and EU ATMP guidelines, Canadian GMP (C.002) and U.S. FDA, PHS Act section 351/361 and CFR 21 Parts 210/211 and Part 11 as they apply to cell and gene therapy. Other guidelines are considered, as required, such as ISO 9001:2015 certification.

4. In-House Versus Outsource: A Decision-Making Guide 5/2/2018

   Biopharma executives must consider how outsourcing fits into their development and production strategy. Here’s a step-by-step guide to help make that decision.

5. Process Development For Scaled-up hESC/hiPSC Manufacturing 11/27/2019

   A manufacturer faced a challenge to scale up, close and optimize a suspension-based manufacturing workflow for pluripotent stem cells (PSCs). An optimized 14-day manufacturing workflow which produces >10¹⁰, cells was developed in < 12 months to create the desired outcomes.

6. Six Dosing And Safety Considerations In The Era Of Emerging Therapies 12/26/2018

   Key considerations for developing early-phase trials that can more accurately define the recommended dose and identify adverse events for emerging therapies.

7. Droplet Digital Polymerase Chain Reaction 1/15/2020

   Leveraging the hyper accuracy of Droplet Digital PCR (ddPCR), viral and gene therapy developers gain additional confidence in tittering assays and vector copy number determination.

8. Commercial Scale Manufacturing of Allogeneic Cell Therapy 11/26/2019

   Allogeneic cell therapy products are generating encouraging clinical and pre-clinical results. Many of these therapies are also expected to have large market sizes and require cell doses of ≥10⁹ cells. As therapeutic technologies mature, it is essential for the cell manufacturing industry to correspondingly develop to adequately support commercial scale production. To that end, there is much that can be learned and adapted from traditional manufacturing fields. In this review, we highlight key areas of allogeneic cell therapy manufacturing, identify current gaps, and discuss strategies for integrating new solutions.

9. Cell Therapy Manufacturing Under GMP - How And When To Get Started 5/2/2019

   Emerging cellular therapies aim to manufacture cell populations for clinical indications, such as cancer, autoimmune, and cardiovascular diseases. As hospitals and translational facilities explore implementation of this technology they concurrently evaluate whether or not to manufacture the therapies onsite. Here is a guideline of how and when to get started.

10. How To Speed Up Cell And Gene Therapy Treatments For Patients, Reduce The Number Of Manual Operations And Eliminate Operator Errors 10/9/2019

    This white paper addresses the unique production problems of cell and gene therapy and explains how a manufacturing execution system (MES) can address them. It provides guidance on how to select an MES and a case study of two CAR-T therapy companies scaling up with and without an MES.