FDA Seeking to Enhance Manufacturing of Cell Therapy and Gene Therapy Products

More consistent and reliable production cell therapy and gene therapy processes are critical for advancing innovative treatments.

While cell therapy and gene therapy products hold the promise of transforming the treatment of many diseases, difficulties in achieving consistent process control could stymie advancement in this field, cautions Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER). It’s “not simple” to bring a clinical production process to commercial scale, he says, voicing fears that important manufacturing issues could hinder efforts to advance new discoveries able to transform medical care.

With more than 100 investigational new drug applications (INDs) filed last year for gene-therapy products and multiple R&D programs underway, FDA seeks to emphasize the challenges of developing and validating relevant manufacturing processes. CBER is receiving dozens of applications to test new gene therapies, Marks reported at the Cell and Gene Therapy Symposium in July 2018, sponsored by CASSS. He acknowledged that the logistics of manufacturing autologous cells can be particularly challenging, and that limited capacity in cell lines stymie efforts to meet the demand for product development. While there is great excitement over the potential of CAR-T (chimeric antigen receptor) cell therapy to target and activate T cells to eliminate a cancerous or other undesirable cell types, Marks cautioned that the use of genetically modified cellular therapies to achieve a durable therapeutic effect requires a consistent process for producing and characterizing cells.

Marks suggested that new manufacturing technology, such as partially automated closed systems and…continue reading article.