The first CRISPR-Cas9 based therapeutics are now in Phase I/II clinical trials, such as the treatment of sickle cell disease developed by CRISPR Therapeutics and Vertex. Regulations and guidelines valid for cell and gene therapies also apply for CRISPR-mediated therapies (i.e. Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs): Guidance to Industry) published by FDA.