Skip to Content

Expanded Access Policy

Expanded Access Policy

Intellia is committed to solving the complex challenges of making CRISPR-based therapies a reality for patients suffering with severe and life-threatening genetic diseases. In these efforts, Intellia is guided by ethical, scientific and legal principles, which require well-designed and controlled clinical trials to evaluate the safety and efficacy of our potential therapies. Visit the National Institutes of Health (NIH) website to learn more about clinical trials and how they work.

Under certain circumstances, a person suffering a serious or life-threatening disease may ask to use an experimental treatment outside a clinical trial before its safety and efficacy have been fully evaluated, and before the regulatory authorities have approved it. Generally, this is an option only for patients who have exhausted all available medical options and do not qualify for ongoing clinical trials. The U.S. Food and Drug Administration (FDA) explains such “expanded access” as follows:

Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Intellia appreciates the intent of expanded access programs but, at this time, we do not provide access to our investigational therapies outside of clinical trials. In our view, we can best advance the development of these potential promising products by enrolling patients in clinical trials. Our clinical trials are designed, conducted and monitored to ensure that the safety and effectiveness of our experimental therapies are properly evaluated before they are made available generally.  To view a list of Intellia’s open clinical trials, visit

As the pace of innovation advances, particularly in the field of gene editing, Intellia is committed to regularly reviewing, and updating as needed, its policy on expanded access.  As part of this pledge, a committee of senior leaders at Intellia has been established to assess both the progress of our clinical programs as well as any changes in the treatment landscape and external environment that may necessitate an adjustment to our expanded access policy. Additionally, we consult with patient organizations, external bioethicists and clinical experts to help inform our expanded access approach. Through this work, we look to balance immediate patient needs with our ability to conduct clinical trials and address the logistical and technical considerations of providing broader access to our investigational gene editing medicines.

Should you have any questions, please reach out to [email protected] or speak with your physician. Intellia expects to acknowledge receipt of requests sent to this email address within five business days.

As authorized by and in accordance with the 21st Century Cures Act, Intellia reserves the right to revise this policy at any time.