CRISPR technology has been used to successfully treat sickle cell disease, and now, the CRISPR gene-editing tool has been shown to work safely and effectively when used inside the patient’s body, or ‘in vivo,’ for the first time.
CRISPR conducts cut-and-paste edits to the genome inside cells, but this process usually takes place outside of the body before cells are returned to the patient. This is the first time the process has been conducted entirely within patients.
The phase 1 trial, conducted by Intellia Therapeutics and Regeneron Pharmaceuticals, combats transthyretin amyloidosis, a disease that causes a patient to make excessive proteins that can gather on nerves and organs, leading to pain and complications which can be fatal.
The use of CRISPR in this trial was shown to be more effective than existing treatments with no serious side effects. The trial consisted of six patients, with plans to expand to a larger test group during phase 2 of the trial next year.
Source study: Intellia Therapeutics – Intellia and Regeneron Announce Landmark ClinicalData Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA- 2001
