One biotech company reported that medically, injecting the CRISPR gene editor into the bloodstream of three patients with a rare genetic disorder could alleviate their symptoms
Data reported at the conference today in Berlin for this disease, called hereditary angioedema
Fyodor Urnov, a CRISPR researcher at the University of California, Berkeley, said the clinical data was "impressive" and "an important achievement in the field
CRISPR has been shown to treat blood disorders through an in vitro strategy that collects a patient's cells, makes them edited in the lab, and then returns them to the patient, in addition, a gene editor is injected into the eye, also showing temporary benefits
Last year, Intellia and partner Regeneron reported in a landmark study that it had a rare genetic disorder called transthyroxine (ATR) amyloidosis, a CRISPR drug in the body that blocks the accumulation of liver proteins that can cause nerve pain, numbness and heart problems
The disease is caused by mutations in a protein called C1 esterase inhibitors, which are part of a signaling pathway that controls bradykinin levels, a peptide hormone
Drugs can help prevent these attacks
To implant the gene editor in vivo, the company wrapped the guide RNA and messenger RNA encoding the enzyme in lipid nanoparticles
Clinical immunologist Hilary Longhurst reported at the 2022 Bradykinin Symposium that in the Hereditary Angioedema Trial, three patients treated with low-dose CRISPR had an average of 65% decline in kallikrein over 8 weeks.
In three patients recently treated with higher doses of CRISPR nanoparticles, kallikrein levels dropped even more, by 92 percent
The team's patients now have no seizures, and patients report that CRISPR treatment has "changed lives" and they no longer worry about strenuous physical activity or swelling episodes
Other teams are also testing in vivo gene editing to treat various diseases
