Three people with a rare genetic disease are being helped by an injection of the CRISPR gene editor. The treatment was able to stop the swelling of the throat and limbs caused by aLiverProtein that causes painful and potentially life threatening bouts ofSwelling in the throat and limbs. Two people in the company's trial are doing so well that they no longer need drugs.
The data was reported at a meeting in Berlin. In the past, the company has used in-vivo delivery of CRISPR to inactivate genes in a person. The first report of clinical benefits associated with injecting the tool is reflected in the latest results.
The data is impressive and an important achievement for the field, says a researcher.
A patient's cells are edited in a lab in order to treat a blood disorder. It is possible to inject a gene editor into the eye to treat blindness disorders. It's more difficult to direct CRISPR to specific organs or cells via an IV.
Last year, in a landmark study, Intellia and partner Regeneron reported that in people with a rare genetic disease called transthyretin (ATTR) amyloidosis, an in-vivo CRISPR drug halted the build-up of liver genes that can cause nerve pain, numbness, and The company hasn't yet revealed if the patients' symptoms improved after they were knocked down. The benefits emerged quickly for the trial.
The disease is caused by the disabling of a signaling pathway that controls bradykinin, a hormone that causes blood vessels to leak fluid. Severe swelling of limbs, the abdomen, or the throat can be caused by high blood levels of bradykinin, which can be triggered by stress, illness, or trauma.
Drugs can help prevent these attacks by blocking kallikrein, which causes bradykinin levels to go up. Patients could avoid using those drugs for the rest of their lives by permanently disabling the kallikrein genes. In order to take on that challenge, Intellia used a strand ofRNA that guides the genes to be cut.
In order to deliver the gene editor in the body, the company wrapped the guide and messengerRNAs together. When kallikrein is injected into a patient's bloodstream, the particles go to the liver, where they are taken up by the cells there. The genes for kallikrein are ferried to and snipped with the help of the CRISPR.
Hilary Longhurst reported at the Bradykinin Symposium that three patients who received a low dose of the CRISPR treatment saw their kallikrein blood levels drop by 65 percent on average. Longhurst is the leader of the New Zealand arm. After 10 weeks, a third of the people who were having up to seven attacks a month stopped.
Two of the three were taking drugs. The attacks stopped after they went off the meds.
Three patients with a higher dose of CRISPR had their kalakrein levels drop by more than 90 percent. Leonard says the results suggest the company's early success is not a fluke.
The treatment has changed the lives of many of Longhurst's patients. They don't worry that a hard gym workout or catching a cold will cause a swelling attack. She says it's incredible to have someone cured of symptoms for the rest of their lives.
Other teams are trying to find a cure for many diseases. In July of this year, the company Verve began a clinical study of base editing for an inherited form of high cholesterol. Verve's drug swaps one DNA base in its sequence for another, a potentially safer method of disabling a problematic gene.
All Rights Reserved © 2024
