Research Report ‘Can new gene therapies result in unintended germline modification?’

Germline modification involves changes to germ cells, which can have (adverse) consequences for offspring. Modifying germ cells is not permitted. It is therefore of great importance to know whether unintended germline modification could occur as a ‘side effect’ of gene therapy, and what consequences this might have if treated individuals were to have children in the future.

The Central Committee on Research Involving Human Subjects (@CCMO) and the Commission on Genetic Modification (COGEM) commissioned a study to assess the risk of unintended germline modification, particularly in relation to new forms of gene therapy. The study was conducted by Sciensano and Perseus BV.

The report examines the various forms of gene therapy currently under development and provides an update on the available data regarding the distribution of gene therapy throughout the body (biodistribution) and the likelihood of unintended germline modification occurring. It also summarises the relevant gene therapy guidelines issued by bodies such as the FDA and the EMA.