Decentralized manufacturing: from stem cell transplants to the next generation of cellular immunotherapies

Published: 18 June 2020
Expert Insight
Matthew Li,
Matthew Li
BioMedical Scientist & Engineer at Vor Biopharma
Matthew Li is Biomedical Scientist and Engineer with over 10 years of experience across an array of areas in the biotech field including CGT R&D and manufacturing, medical device development, and clinical research. He currently leads the Cell Therapy Manufacturing Innovation group at Vor Bio that engages across the CGT value chain interfacing with research, development, manufacturing, quality, regulatory, clinical, business development, and commercial to holistically address and solve challenges across numerous stages of development and production. Matthew holds a PhD in Medical Engineering and Medical Physics from the Massachusetts Institute of Technology.
Sadik Kassim
Sadik Kassim
CTO at Vor Biopharma
Dr. Sadik Kassim is Executive Director at Kite Pharma, where he oversees the CMC activities for all next generation immune cell therapies for cancer. Previously, Sadik was Chief Scientific Officer of Mustang Bio. Prior to Mustang, Sadik was Head of Analytical Development for Novartis’ Cell and Gene Therapies Unit in Cambridge, MA where he contributed the BLA and MAA filings for Kymriah™. Earlier in his career, Dr. Kassim was a research biologist in the Surgery Branch at the National Cancer Institute, where he was involved in early research and CMC work that led to the development of Kite’s Yescarta™ (CD19 CAR T) for lymphoma. Sadik was a research fellow in the University of Pennsylvania’s (Penn) Gene Therapy Program where he led the initial discovery efforts and pre-clinical studies for an AAV8 gene therapy for familial hypercholesteromia. This program is now in the clinic as part of RegenexBio’s RGX-501 program.

Cell and gene therapies are emerging as pillars of modern medicine. Yet, there remain significant challenges related to drug product manufacturing scale out, accessibility, and overall pricing of these potentially curative therapies. To date, drug developers have pursued more traditional models of centralized manufacturing to enable for the commercial scale out of approved cell and gene therapies. These traditional manufacturing models enable process and product control. The pursuit of a centralized manufacturing model for autologous personalized cell and gene therapies, however, could lead to significant complexity with respect to overall logistics and manufacturing costs. A more distributed model of manufacturing can potentially provide patients with faster access to drug product and lead to greater overall cost savings. A decentralized model, could however, also lead to significantly less process and product control for the drug developer. This brief article will examine the potential path that drug developers can pursue to enable decentralized manufacturing for commercial cell and gene therapies. Specifically, we discuss the existing stem cell transplant center infrastructure in the USA and how it may be leveraged to enable for a more decentralized model of manufacturing.

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