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Hereditary angioedema (HAE) is a rare genetic disorder characterized by severe, recurrent and unpredictable swelling of organs and tissues in the body that can cause pain, debilitating and life-threatening
.
A new study presented this year at the annual scientific meeting of the American Academy of Allergy, Asthma and Immunology (ACAAI) in Louisville, Kentucky, shows that treatments using CRISPR genome editing technology have successfully reduced swelling and reduced the frequency of
attacks.
Clinical immunologist Hilary Longhurst, MD, first author of the study, said: "NTLA-2002 is a one-off, systematic-administered candidate for CRISPR genome editing that is being developed
for HAE.
It is designed to remove the KLKB1 gene from liver cells, thereby reducing the production of a specific protein called klikrein, whose uncontrolled activity is responsible
for relapse, debilitating and potentially fatal episodes of swelling in HAE patients.
”
respectively to date.
Danny Cohn, MD, senior author of the study, said: "Although the data are still increasing, plasma kininase levels decreased rapidly and significantly in all patients in the 25- and 75 mg dose groups treated with a single dose of NTLA-2002
.
In the 25 mg dose cohort, 3 patients who reached a predetermined time point to assess HAE episodes also had a significant reduction
in HAE episodes in all patients.
”
At doses of 25 and 75 mg, NTLA-2002 was well tolerated, with most adverse events of mild severity
.
The most common adverse event was infusion-related reactions, mostly grade 1, which resolved
within a day.
To date, no dose-limiting toxicity, serious adverse events, and grade 3 or higher adverse events
have been observed.
Dr Longhurst said: "These early data support NTLA-2002 as a potential one-time treatment
to cure HAE symptoms.
Clinical development of the project will continue, with randomized Phase II clinical trials beginning
in the first half of 2023.
”
