When and How to Engage with Your Regulator
Whether you are developing a therapeutic or an enabling technology, you know that ambiguity can be one of the biggest hurdles to overcome in early development and can slow down or even stall decision making. One way to reduce ambiguity, and therefore risk, is to consult with regulators on your planned development activities. Companies may engage with Health Canada, U.S. Food and Drug Administration (FDA), European Medicines Association (EMA) or other global regulators as part of this process.
Posted: Jun 8, 2020 11:00:07 AM
Viral Aggregation in Downstream Processing of Lentiviral Vectors
Viral vectors are vehicles for delivery of therapeutic DNA in cell and gene therapies. With over 1,000 cell and gene therapy (CGT) clinical trials underway globally, there is a growing need to address challenges in viral vector manufacturing – both upstream and downstream. In a previous post, we outlined the key steps in downstream processing (DSP) for the manufacturing of lentiviral vectors (LVV). Achieving high concentration without aggregation is a significant technical bottleneck in manufacturing LVV. In this post, we will explore the concept of viral aggregation and its influence in every step of DSP for LVV.
Posted: May 11, 2020 10:57:59 AM
Quality-by-Design Approach to Manufacturing Cell and Gene Therapies
Implementation of a manufacturing process that assures a predefined quality of product is a critical requirement for the licensing and marketing of every cell and gene therapy (CGT) product.
Posted: Apr 17, 2020 2:15:02 PM
Essentials of Auditing in the Manufacturing of Cell and Gene Therapies
Our previous posts on quality management systems and steps to ensure your cell and gene therapy (CGT) product is Good Manufacturing Practices (GMP) compliant alluded to auditing in the context of a larger manufacturing workflow. This post provides useful insights into auditing to help clients and contract manufacturing organizations (CMOs) understand this critical aspect.
Posted: Mar 27, 2020 9:56:27 AM
Three Essential Considerations for Cryopreservation of Cellular Therapies
Cryopreservation helps cells survive both cooling to extreme temperatures and thawing back to physiological conditions. Our previous post covered the importance and challenges involved in cryopreservation of cellular therapies.
Posted: Mar 5, 2020 9:02:27 AM
Understanding Cryopreservation of Cellular Therapies
Cryopreservation is the process of using ultra-low temperatures to preserve living cells and tissues for a prolonged time period.
Posted: Feb 13, 2020 2:13:44 PM
Induced Pluripotent Stem Cells – Part 1: Understanding Reprogramming
Stemming (pun intended) from the fundamental question in developmental biology around whether cellular differentiation could be reversed – like many transformative scientific discoveries – identification of induced pluripotent stem cells (iPSCs) was a curiosity-induced accident. In their foundational 2006 study, Takahashi and Yamanaka determined that fully differentiated adult skin cells could be reprogrammed back into stem cells by the over-expression of four genes (Oct3/4, Sox2, Klf4, c-Myc).
Posted: Jan 29, 2020 1:58:32 PM
Road to success: Understanding Good Laboratory Practice for cell and gene therapies
Good Laboratory Practice (GLP) studies are essential for generating nonclinical study data supporting drug product submissions or applications for human use. According to the most basic definition, GLP is a set of principles that can be applied to ensure the quality and integrity of non-clinical laboratory studies. GLP provides a means to standardize the results of pre-clinical studies that come from different labs, in part by imposing increased documentation requirements based on standard procedures. Standardization is the driving force behind GLP.
Posted: Jan 10, 2020 1:36:17 PM
What Are the Benefits of an Automated High-Throughput Screening Process for Custom Cell Culture Media Development?
We are often asked if culture media development can be customized at the discovery stage of the product, and if feasible, how it can be done. In this post, we will outline one approach that can be considered to customize media development for discovery-based processes.
Posted: Nov 21, 2019 10:59:14 AM
Five Steps to Ensure Your Cell and Gene Therapy Product Is GMP Compliant
When we 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. A lot of complex research and testing is performed to confirm Proof of Concept and that the therapy performs as desired.
Posted: Oct 30, 2019 3:59:26 PM