Marks Suggests Using Animal Models For Gene Therapy Is Undesirable

FDA biologics center chief Peter Marks said the testing of gene editing therapy, specifically CRISPR-Cas9-associated protein 9 (CRISPR-Cas9) in animal models, which some companies have relied on, is “less than ideal” because testing requires guides specific to human DNA. Marks suggested the possible use of organoids and humanized mice as alternate solutions for model systems. “Preclinical in vitro and in vivo studies are recommended to support the scientific rationale and safety for administration of an investigational genome editing product...