Genome editing has been a hot topic in drug discovery research recently, as CRISPR-based preclinical programs inch closer to the clinic this year, joining the clinical-phase ranks of Zinc Finger and TALEN-based therapies. These revolutionary techniques, collectively referred to as “genome editing,” are being investigated as therapies that directly target the underlying root causes of diseases, including many devastating genetic diseases, various cancers, and infectious diseases.
Last month, the Human Genome Editing Initiative of the National Academies of Sciences and Medicine (NAS/NAM) released a Consensus Study on Human Gene Editing, which examined the clinical, ethical, legal, and social implications of these technologies. The report is well over 200 pages long and is the result of the authors’ year-long examination of the issue in public forums before reaching their conclusions.
BIO’s FAQ answers basic questions, such as “What is genome editing?” to more detailed questions surrounding oversight and regulatory frameworks covering the new technology. Examples of disease areas being addressed in current and future clinical programs are also presented.
The FAQ reiterates that BIO member companies – along with the scientific community – are focused on therapeutic applications editing somatic (non-reproductive) cells that will only impact the consenting patient, not their potential offspring. As such, current regulatory processes that are used in gene therapy can be directly applied to genome editing, a point emphasized in the NAS/NAM study and a view shared by the American Society of Gene and Cell Therapies (ASGCT).
For answers to other regulatory questions, or a quick primer on the topic, view BIO’s FAQ on human genome editing.