Published on March 15, the guidance documents cover recommendations including product manufacturing and clinical trial design.
The draft guidance, ‘Human Gene Therapy Products Incorporating Human Genome Editing,’ has been created to guide sponsors developing human gene therapy products incorporating genome editing (GE) of human somatic cells.
It provides recommendations regarding information that should be provided in Investigational New Drug (IND) applications for GE products, including information on product design, product manufacturing, product testing, preclinical safety assessment, and clinical trial design.
Meanwhile, the draft guidance ‘Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products,’ is intended to assist sponsors developing human gene therapy products in which the T cell specificity is genetically modified to enable recognition of a desired target antigen for therapeutic purposes.
It provides CAR T cell-specific recommendations regarding chemistry, manufacturing, and control (CMC), pharmacology and toxicology, and clinical study design.
“While this guidance specifically focuses on CAR T cell products, much of the information and recommendations provided will also be applicable to other genetically modified lymphocyte products, such as CAR Natural Killer (NK) cells or T cell receptor (TCR)‑modified T cells,” notes the FDA.
The guidance can be found via the following links: Human Gene Therapy Products Incorporating Human Genome Editing, and Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products.
