Regenerative medicine evolved from medical research rather than from drug-development companies, and technologies that worked in clinical settings haven’t translated directly to large-scale manufacturing. Cost is often the problem for chimeric antigen receptor (CAR) T-cell therapies. And whereas the classical drug field increasingly is called out for massive mark-ups and huge advertising budgets, the price of regenerative medicines truly is linked directly to the cost of making them. This month’s Featured Report gathers industry perspectives on the status of the “CAR-T conundrum,” exploring what it will take for those promising new therapies to become affordable and accessible to patients. Read on to learn about challenges that remain with CAR-T manufacturing, discussions surrounding the need for distinct regulatory requirements for advanced therapy production, recent developments in CAR-T commercialization, and the escalation of private cell-banking abuse.   

Introduction: An Overview of Challenges and Opportunities in CAR T-Cell Development and Manufacturing by Cheryl Scott
BPI’s senior technical editor begins this in-depth Featured Report by highlighting recent conference talks, then providing further perspective on CAR T-cell manufacturing through interviews with Gabriel Festoc (Polyplus-transfection), Ravi Nalliah (TrakCel), Vered Caplan (Orgenesis), Jenna Balestrini (Draper), Xiuyan Wang (Memorial Sloan Kettering Cancer Center), Benoit Bossuge (Novartis Technical Operations), Philippe Parone (Celyad), and Ping Jin (National Institutes of Health Clinical Center).

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Cell Banking for Cell and Gene Therapy: Regulatory, Ethical, and Scientific Considerations by Laertis Ikonomou, Aaron D. Levine, Peiman Hematti, and Bruce L. Levine
The tangible clinical promise of cell, gene, and tissue therapy has brought about the emergence of tagalong marketing for services both legitimate and dubious, including speculative private human–cell-banking services. By contrast with legitimate cord-blood banks, speculative companies focus on long-term cell storage with an unclear path to clinical translation. In this article, members of the International Society for Cell and Gene Therapy’s (ISCT’s) Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapies explain this trend’s risks for patients as well as developers of CAR T cells and other therapies. Publicly run and funded cord-blood banks could offer a viable alternative.

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Do We Need Separate Regulations for Advanced Therapies? by John R. Godshalk
Talk has circulated recently in the industry about a possible need for separate good manufacturing practice (GMP) regulations to cover cell and gene therapies. Here, the author reviews US and European GMP guidelines — focusing especially on chemistry, manufacturing, and controls (CMC) and facility issues — with the perspective of someone who’s been both a Food and Drug Administration (FDA) reviewer and industry consultant. Godshalk believes that new GMP regulations for CAR T-cell manufacturing are unnecessary, but that official guidance documents would be helpful both to companies putting marketing applications together and to the regulators who review those applications.

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CAR-T in the News: A 2019 Wrap-Up from BioProcess Insider by Dan Stanton
BioProcess Insider editor Dan Stanton highlights results from Informa’s “Cell Therapy Analytics” report and a recent BPI Conference presentation from Kite Pharma touting the allogeneic approach to CAR T-cell manufacturing. Kite’s senior vice president and global head of manufacturing emphasizes a number of requirements for taking this therapeutic modality to the next level: operational efficiencies for improved patient access, scaling out geographies and capabilities through collaborations both within the industry and externally with healthcare providers, and digital and “industry 4.0” capabilities. Finally, an interview with Miguel Forte (former Zelluna Immunotherapy CEO) introduces a possible alternative approach to CAR T-cell manufacturing for solid tumors.

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