Expert Insight

Optimizing the Cell Collection Network to Support the Commercialization of Cell & Gene Therapies

Spotlight Article

Interview

Amy Hines, Kimberly Kasow, Sandra Hoffmann

Raw Materials for Cell & Gene Therapy: Getting it Right from the Start

Amy Hines, Be the Match

Amy Hines BSN, RN, is the Director of Collection Network Management for Be The Match BioTherapies®. She oversees the performance of apheresis centers and cell therapy labs in the Network, and ensures their ongoing compliance with FDA and international standards and criteria, industry best practices, and appropriate regulatory and accrediting entities. She joined Be The Match BioTherapies’ parent company, the National Marrow Donor Program® (NMDP)/Be The Match®, in 2013 managing the NMDP/Be The Match apheresis and collection center Network of more than 90 apheresis centers and 80 collection centers. Amy has nearly 20 years of experience in the cellular therapy field, starting her career as a stem cell transplant registered nurse. She received her Bachelor of Science in Nursing from Grand Valley State University in Allendale, Mich.

Dr Kasow, Be the Match

Kimberly Kasow, DO, is the Director of the Pediatric Bone Marrow Transplant program at the University of North Carolina (UNC) at Chapel Hill. In addition, she is the Associate Director of BMT CTP Quality Initiatives, the Bone Marrow Collections Medical Director and a Clinical Professor in the Department of Pediatrics, Division of Hematology/Oncology. Dr. Kasow completed her pediatric residency at Columbus Children’s Hospital and trained as a fellow in the Department of Hematology/Oncology and Division of Stem Cell Transplantation at St. Jude Children’s Research Hospital. Dr. Kasow works closely with the UNC adult and pediatric transplant clinical programs, the apheresis facility and the HPC processing facility to ensure they perform as a unified cellular therapy program. She is dedicated to maintaining a high-quality program to ensure better patient outcomes and bringing new cellular therapy options to patients.

Sandra Hoffmann, MT (ASCP) SSB, CQA (ASQ), is the Supervisor of the Cellular Therapy Laboratory at the University of Michigan. She oversees all aspects of HPC processing, cryopreservation and distribution, and ensures compliance with all regulatory requirements associated with HPC components. As a Certified Quality Auditor (CQA) per the American Society for Quality (ASQ), Sandra performs internal and external audits. She has co-authored more than a dozen publications. Sandra has more than 25 years of experience in the clinical laboratory setting. She joined the University of Michigan as a Clinical Laboratory Scientist in 1993, and started her career as a medical technologist in blood banking. Sandra received her Bachelor of Science in Medical Technology from Wayne State University College of Allied Health and Pharmacy in Detroit, Mich.

DOI: 10.18609/cgti.2018.021
Citation: Cell Gene Therapy Insights 2018; 4(3), 197-206.
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Making the grade: untangling the myths of raw materials used for the manufacture of cell- & gene-based medicinal products

Spotlight Article

Expert Insight

Christopher A Bravery, Sarah Robinson & Scott R Burger

SUPPLY CHAIN MANAGEMENT PART 1: GETTING IT RIGHT FROM THE START

Cell and gene therapy medicinal products (CGP), like other medicines for human use, are expected to be consistently manufactured to a defined quality. That quality is demonstrated through preclinical and clinical studies to be suitable based on an overall assessment of the risks (including quality) and benefits of the product when used to treat a medical condition. Achieving a consistent quality product requires an overall manufacturing control strategy including control of materials, control of the process, control of any intermediates, drug substance and final drug product. Consequently the quality of materials, including raw materials, used to manufacture are paramount to the final CGP quality. This review aims to cut through various mythologies around raw materials by taking a regulatory science approach to discuss raw materials selection.

Submitted for peer review: Apr 1 2018 Published: Apr 5 2018
DOI: 10.18609/cgti.2018.022
Citation: Cell Gene Therapy Insights 2018; 4(3), 207-225.
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Addressing Particulates, Extractables & Leachables & the Quality of Single-Use Systems for Cell & Gene Therapy Manufacturing

Spotlight Article

Innovator Insight

Dominic Clarke

SUPPLY CHAIN MANAGEMENT PART 1: GETTING IT RIGHT FROM THE START

Cell and gene therapies (CGT) continue to demonstrate great promise and the recent commercial successes over the past year have further strengthened excitement and support for the industry. While improvements and advancements continue, manufacturing of CGTs continues to offer challenges. Experiencing manufacturing challenges is expected with an emerging industry, but manufacturing CGTs also presents some unique complexities when compared to similar industries. Production of CGTs, and specifically patient-specific products, rely exclusively on single-use systems (SUS) as the raw materials. Since the product is the output of the process, the quality of the raw materials used in the manufacturing process is critical. CGT raw material quality includes a wide range of aspects, many of which require specific attention given the heavy interaction of the cells and the SUS. For the context of this paper, the main focus will center on the growing need to further understand how to address particulate matter and extractables and leachables for CGT products.

Submitted for peer review: Feb 5 2018 Published: Apr 5 2018
DOI: 10.18609/cgti.2018.020
Citation: Cell Gene Therapy Insights 2018; 4(3), 187-196.
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Understanding the Role of Critical Quality Attributes along the Commercialization Pathway

Spotlight Article

Interview

Anna Gilbert

Raw Materials for Cell & Gene Therapy: Getting it Right from the Start

 

Anna Gilbert has over 20 years of experience in Quality Assurance in Biotechnology, with the last 8 years focused in cell therapy. Anna is currently the Director of Quality at Nohla Therapeutics, Inc. Prior to Nohla, Anna has prior experience at several biotech companies. Most recently she started in January 2014 at Juno Therapeutics as one of the earliest employees and the first in Quality Assurance. She established the first quality systems for Juno and managed the GMP quality compliance program. At Dendreon, Anna managed all aspects of GMP and GTP quality compliance, including internal and external auditing as well as compliance oversight of apheresis sites. She joined Dendreon in 2009 and participated in the successful pre-approval inspection of the first US commercial cell therapy, Provenge. Anna is an ASQ Certified Quality Auditor and a member of the Parenteral Drug Association (PDA). She delivers an annual lecture on the topic of GMP
auditing at University of Washington’s Master of Science in Biomedical Regulatory Affairs program.

DOI: 10.18609/cgti.2018.023
Citation: Cell Gene Therapy Insights 2018; 4(3), 227-230.
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The Evolution of Product Transfer and Labeling in Cellular Therapy

Spotlight Article

Innovator Insight

Lizette Caballero

Raw Materials for Cell & Gene Therapy: Getting it Right from the Start

Lizette Caballero works at the University of San Francisco California where she serves as the Senior Laboratory Supervisor overseeing the Blood and Marrow Transplant Laboratory. Lizette has over 20 years’ experience working in the field of cellular therapy. She has run the BMT labs at Florida Hospital in Orlando and at UCSF. Her experience includes conducting research and development of new instruments and techniques, development and revision of laboratory procedures, compiling quality control data and managing and training employees. Lizette is very active in the cell therapy section of AABB and ISCT. She has presented many times on a variety of topics and has published numerous papers. Her work includes studying the synergistic cytotoxicity of IFN-2b and IL-2 in combination with PMBC against ovarian cancer and studying the effect of MSCs for treatment of acute respiratory distress syndrome. For the last 15 years she has also served as a FACT Inspector for cellular therapy processing facilities having completed over 20 inspections.

DOI: 10.18609/cgti.2018.007
Citation: Cell Gene Therapy Insights 2018; 4(3), 51-57.
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Innovation in Management as a Tool for Accelerated Translation of Advanced Therapies

Spotlight Article

Expert Insight

Nicholas Medcalf

SUPPLY CHAIN MANAGEMENT PART 1: GETTING IT RIGHT FROM THE START

The manufacture, delivery and administration of advanced therapies (cell-, tissue- and gene-based treatments) comprise a sensitive chain of custody that may be thought of as an integrated service system. The system is challenging to assemble for the predominantly small businesses that seek to compete in this sector. The current investment climate is not favorable to proposals unless they have a high probability of success or are capital-efficient or both. Business models based around the extended enterprise, the collaborative enterprise and the franchise models are relatively under-explored for the sector. This paper sets out the main characteristics of these models and the reasons that they may increase the pace of innovation: by reducing investment burden, by encouraging co-ownership of the chain of custody and by allowing early data capture to assist with investment proposals.

Submitted for peer review: Dec 15 2017 Published: Apr 5 2018
DOI: 10.18609/cgti.2018.011
Citation: Cell Gene Therapy Insights 2018; 4(3), 157-172.

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