Clinical development programs for human gene therapy products that incorporate genome editing should address the potential risks of off-target editing as well as unintended consequences of on-target effects, agency says in new draft guidance.
The US Food and Drug Administration wants developers of gene therapy products that incorporate human genome editing to pay special attention to safety issues resulting from both on- and off-target effects of editing.
In a new draft guidance, the FDA recommends that clinical development programs for human genome editing (GE) products address...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
