Clinical application of AAV vectors containing targeted nucleases such as CRISPR-Cas9

COGEM was asked to advise on whether or not the use of targeted nucleases, such as CRISPR-Cas9, in AAV vectors in clinical trials meets the requirements of the COGEM generic environmental risk assessment for AAV vectors, and on the need for additional containment measures. COGEM was also asked if its answer also applies to future gene-editing applications in combination with AAV vectors. COGEM advises that clinical applications involving AAV vectors containing targeted nucleases should be subject to a further condition requiring additional hygiene measures to be taken after administering the AAV vector. This condition may be waived if an applicant provides a sound argument for doing so