Gene Editing Stakeholder Comments

Lonza Biologics says it appreciates FDA’s efforts to make available the agency’s current thinking on a range of topics applicable to the development of cell and gene therapy products. “Lonza particularly appreciates information specific to chemistry, manufacturing, and controls (CMC) expectations,” the letter says. The company includes detailed specific comments on the CMC section.

Pharmaceutical Research and Manufacturers of America (PhRMA) says the agency’s recommendations are essential in helping sponsors navigate issues in this rapidly evolving field. Its comment letter identifies areas in the docket that it says would benefit from additional clarification.

Bayer’s comment says the guidance addresses the definition of in vivo gene editing components very well but does not address the definition of gene editing components when used ex vivo.

Specific line-by-line comments were submitted by Pfizer, the International Society for Pharmaceutical Engineering, and the Parenteral Drug Association.