Newsletter | October 1, 2019

10.01.19 -- Limitations & Opportunities Of CAR T-Cell Therapies


Hello Cell & Gene readers,


A few weeks ago, I shared with you my article, The Three Pillars of Patient Advocacy. In that article, I promised forthcoming feature editorial that would take a deep dive into why patient advocacy is a crucial component in the ongoing treatment of rare diseases. Well, it’s here; read on for my conversation with Jamie Ring, head of patient advocacy at Spark Therapeutics, about the role of patient advocacy in gene therapy.


CAR T cells show immense potential, but they also face substantial challenges to more widespread adoption. Here’s an in-depth look at the current limitations and future opportunities of CAR T Cell therapies.


Finally, I’m attending ARM’s Meeting on the Mesa this week. I like to meet our readers in person and to learn about their roles in the cell and gene sector. Email me or Tweet me if you’d like to connect at the conference. I hope to see you there!  


Erin Harris, editor in chief
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Industry Insights
Overcoming The Unique Filling Challenges Of Autologous Therapies
Article | By Herman F. Bozenhardt and Erich H. Bozenhardt, Integrated Project Services (IPS)

Autologous (patient-derived) therapies represent unique challenges to processing. One-patient, single-batch is a radical change from the scale our industry is accustomed to. At this level the filling is more analogous to limited Phase 1 clinical trial materials or even preclinical applications. The logistics and required compliance of manufacturing these personalized therapies is drastically different and presents a combinatorial explosive problem. One aspect of this process where traditional methods fall apart is filling of these therapies.

Cell And Gene Therapies: SUT Connection Lessons From Biotech
Article | By Derek Pendlebury, CPC

Single-use connection technologies are expected to play a critical role in commercial manufacturing of cell and gene therapies. Learn about the types of connection technologies and selection considerations.

Moving Toward A Closed CAR T Cell Manufacturing Process
Poster | By Devina Ramsaroop, Steven Loo Yong Kee, Danylo Sirskyj, Calley Hirsch, Elizabeth Csaszar, and Aaron Dulgar Tulloch, GE Healthcare Life Sciences

Challenges to overcome in the CAR T manufacturing process include lengthy production times (11 -  21 days), product variability associated with open handling steps, and high manufacturing costs. Here we examine and evaluate individual CAR T cell unit operations, commercial reagents, and equipment with process closure potential to develop an improved workflow and increased product consistency.

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