Newsletter | November 17, 2020

11.17.20 -- CAR-T & The Voice Of The Patient: An Update

From The Editor

Greetings Cell & Gene readers,


Establishing a policy of required GDP/DI for all employees from the outset is cheap insurance for the future of your projects. Here’s how.


Back in 2018, I shared with you the CAR-T journey of friend of Cell & Gene, Bob Levis. Bob is now the director of the CLL Society and a Cell & Gene Editorial Advisory Board member. In addition to his work with the CLL Society, he keeps busy with the Penn Medicine Leadership Council. Here, Bob shares his thoughtful update on his personal patient perspective and experience with CLL.


Erin Harris, editor in chief
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Industry Insights
Understanding The CMC Regulatory Landscape For Cell And Gene Therapy Products

The curing potential of cell and gene therapies is driving the industry to gain a better understanding of the evolving regulatory guidelines for these products, in order to bring them to market faster.

Overcoming Clinical Data Aggregation Challenges
Learn about the challenges for sponsors and CROs when integrating a vast array of data sources and typical relational database structures, why they don’t work for clinical analytics, as well as the ideal clinical data aggregation solutions for continuous, automatic data consolidation.
L-Cystine Peptides Boost Cys-Solubility And Performance In Cell Culture Media Applications

L-Cysteine and its oxidized form L-Cystine are essential amino acids used in cell culture and are present in all chemically defined media formulations. However, while the solubility of free L-Cysteine is high at neutral pH, the applicable concentration in basal and feed media is limited. Read more about an available solution for solubility and performance challenges.

Solutions To Today’s Biomanufacturing Challenges

With more complex molecules in development, changing capacity needs, uncertain forecasts, and increased competition, the market demands flexibility and innovative approaches to today’s challenges.

Designing The Cell Therapy Facility Of The Future

In 1931, Swiss doctor Paul Niehans injected a preparation of live cells from a parathyroid gland into a dying patient. The patient subsequently recovered, and Dr. Niehans had a eureka moment — that injections of living cells can have tremendous therapeutic value.

5 Tips To Avoid An IND Clinical Hold: Regulatory Guidance And Insights

A clinical hold from the U.S. Food and Drug Administration can significantly prolong the time and increase the cost of drug development, which is particularly concerning for emerging/small biotech and specialty pharma companies. In this blog, we discuss common reasons for clinical holds and provide useful tips for both avoiding and addressing them.

Developing Tumor-Infiltrating Lymphocyte (TIL) Therapies: Sample Collection And Processing

While it has been advanced over many years, the process of developing TIL therapies still suffers from certain challenges. This article attempts to lay bare some of these challenges and discuss how novel technological solutions can help overcome them.

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

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.

FDA Requirements For Packaging Cryogenically Frozen Advanced Therapies

The FDA requires packaging and distribution systems to protect the agent from contamination and damage. In distributing cryogenically frozen advanced therapies, this includes protection from temperature shock.

Key Factors For Successful Technology Transfer

Successful technology transfer is reliant on a few key variables including robust process characterization and communication and collaboration among multiple teams and sites. 

Practical Tips To Develop A Lentivirus Packaging Cell Line

CAR-T therapies are expensive, and lentiviral vectors (LV) production represents a large portion of these costs. High manufacturing costs for LV are driven by low efficiencies from limited scalability in adherent cell culture, as well as requirements for large manufacturing spaces and several manual manipulations that increase labor costs. Following are practical tips and a case study to develop a lentivirus packaging cell line.

Strategies To Optimize Virtual And Decentralized Study Designs

Understanding how to incorporate technologies and strategies that decrease the reliance on brick and mortar sites and bring the study to the patient is critical across all phases of drug development in today’s challenging landscape.

Process Development For Scaled-Up hESC/hiPSC Manufacturing

A manufacturer faced a challenge to scale up, close, and optimize a suspension-based manufacturing workflow for pluripotent stem cells (PSCs). In response, CCRM developed an optimized 14-day manufacturing workflow, which produces >1010 cells, in < 12 months.

Full Service Support For A Gene Therapy Trial

A clinical-stage biotechnology startup asked Veristat to run complex U.S. and EU trials of their unique gene therapy. Veristat brought invaluable global capabilities and expertise to a small client team tackling their first clinical-stage program without the large infrastructure or resources to conduct studies in this space themselves. Our end-to-end support started at Phase 1 and will continue into post-marketing and regulatory follow-up.

Reducing Risks With Single-Sourced Modular Cleanrooms

Life sciences industries require highly controlled environments for drug research development and manufacturing.

Effectively Securing Cell And Gene Therapies With Closed Systems

Are you ready to take your therapies from the lab into production and looking for ways to implement a closed system design with single-use technologies?

Process Development For Cell Therapy And Viral Gene Therapy

Process development is a critical enabler to bring safe, effective, sustainable products to market to address patient needs. In this webinar we will discuss our strategies for developing lentivirus and adeno associated virus (AAV) and the impact early decisions can have on commercial readiness.