Ethical Challenges of Gene Editing in Biomedical Research and Healthcare: Patient Autonomy, Safety, Equity and Governance

Gene editing is a reality in clinical practice. Since late 2023 we have seen the approval of an ex vivo CRISPR-Cas9 therapy for sickle cell disease and transfusion dependent beta thalassaemia and in July 2026 the US expanded the label to include children as young as two years of age; also in 2025 a personalized in vivo base editing therapy was made available to an infant; heritable editing is still off the table in most which have a policy against it. We look at four ethical issues which arise from this consent and autonomy, safety, equity and governance which we separate out into categories of somatic, personal and heritable editing and research as opposed to care. We also report that (1) which informed consents were given in past gene transfer studies was in fact to present a greater benefit than what was achieved and in exa-cel we note that the primary serious issues including that of infertility and a fatal liver issue in a child came from the treatment regimen which at the time was under chemotherapeutic conditioning, hence consents had to cover the full treatment process; (2) while regulators at the time found the benefit risk ratio for ex vivo to be favorable they did so based on a 15 year follow up which in in vivo editing we see to have caused a serious liver issue also which in human embryo studies we see to also present a case of chromosome loss which in turn supports the case for licensed somatic use and the to continue to put on hold heritable use; (3) what we do see is that access is the issue not efficacy, we had 64 infusions world wide in 2025 which is against a background of tens of thousands that are eligible and also that the majority of people with SCD live in sub-Saharan Africa; and (4) governance is firm on heritable editing and uneven elsewhere, with public engagement recommended more often than connected to decisions. The literature is concentrated on SCD and exa-cel, and we identify priority evidence gaps.