Cell and Gene Therapy: What’s Hot in 2025

To meaningfully grow, the C&G field needs to build repeatable gene editing platforms and industrialize production 

Because the causes of genetic diseases are so specific, the gene therapies being designed to treat them must be unique as well. This has presented logistical challenges for companies seeking to develop  products for patients with rare and especially ultra-rare diseases, particularly companies with limited capitol. But there are lessons to be learned from the pioneering work done in gene therapies in recent decades, and one of them is leveraging what we already know to create repeatable gene therapy platforms that have the ability to develop gene therapy products with minimal differences. Such platforms remove the burden for every drug by demonstrating consistency on key parameters across all products, building in efficiencies, avoiding certain studies, and enjoying a more streamlined regulatory process. These platforms need ways of accessing and sharing information in order to validate performance of these complex products. And we need companies that can partner with gene therapy companies to help make their products as efficiently and cost-efficiently as possible.

For instance, the US Food and Drug Administration’s Advanced Manufacturing (AMT) Technologies Designation, which our company has, is intended to facilitate the development of drugs made using continuous manufacturing, decentralized manufacturing, 3D printing and other AMTs that improve manufacturing efficiency, reduce production costs and reduce environmental footprints. Look in 2025 for more collaborations and partnerships in creating repeatable gene platforms that can help bring products to more patients more quickly.

The industrialization of C&G products

The industry has shown we can get C&G therapies to work in the clinic and we know we can get them to commercialization. According to the American Society of Cell and Gene Therapy, there are close to 3 dozen cell and gene therapies that have been approved globally, including the first CRISPR-generated therapy (for sickle cell anemia), the first gene therapy for a rare form of blindness and the first cell therapy for multiple myeloma. The next step needs to be industrialization of cell and gene therapies in order to get broader market access and reimbursement. A recent survey cited by Medscape shows that the median time on a waiting list for a life-changing CAR-T therapy was six months, with only 25% of patients eventually receiving one. In short, therapeutic demand is outstripping what companies marketing these products can reasonably accommodate. The National Academy of Sciences reported this year that since 2018 the number of regenerative medicine therapy product developers has increased from 900 to at least 2,700 globally, and that the number of gene and cell therapy clinical trials has increased from around 1,000 to 1,600; the number of patients treated with CAR-T therapies alone has increased from at least 180 to 20,000. Finding methods that enable faster scaleup of therapies will not only help more patients but likely drive the cost of these pricey drugs down. Some of these faster methods could include using common processing methods, building flexible facilities, and ensuring you have hardware platforms that are modular, flexible, integrated, and automated. We are already seeing movement toward greater industrialization, but as the C&G market grows look for full-on industrialization of these therapies throughout the field. 

—Matt Hewitt, VP and Technical Officer for Manufacturing, Charles River