Intellia is committed to developing genome editing treatments for those suffering from severe and life-threatening genetic diseases. We are currently investigating our CRISPR-based technology in clinical trials as single-dose treatments.

Sponsored by Intellia

Transthyretin (ATTR) Amyloidosis

  • Phase 1 Study of NTLA-2001 in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy and Transthyretin Amyloidosis-Related Cardiomyopathy. Learn more on
  • Long-Term Follow-Up Study of Subjects Dosed with NTLA-2001. Learn more on

Hereditary Angioedema

  • Phase 1/2 Study of NTLA-2002 in Adults with Hereditary Angioedema. Learn more on

Participation in a clinical trial is a decision that is made between a patient, their treating physician and the clinical trial site investigator. If you are interested in joining one of our trials, please consult with your physician.

Expanded Access Policy

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. Intellia appreciates the intent of expanded access programs but, at this time, we can best advance the development of these potential promising products by enrolling patients in clinical trials.

Read our full expanded access policy.