New Guidances Say Gene Therapies Require Strong Preclinical Testing

Strong nonclinical testing should underpin development programs for both gene editing (GE) products and chimeric antigen receptor (CAR-T) cellular products, according to two new final guidances issued by the FDA.

Since both of these therapeutics involve genetic alterations of living human cells and potentially, the introduction of viral vectors, sponsors need a firm foundation of in vitro and in vivo testing showing that they are safe and exert limited off-target activity, both guidances say.

CAR-T for non-oncology indications is not covered. The guidance “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products” focuses on the manufacturing and characteristics of CAR-T cells for oncology indications, including hematologic malignancies and solid tumors.

Gene editing products are complex products that can include the gene editor, DNA targeting elements and a donor DNA template, according to “Human Gene Therapy Products Incorporating Human Genome Editing.”

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