CRISPR genome editing treatment relieves swelling attacks in patients with hereditary angioedema

CRISPR genome editing treatment relieves swelling attacks in patients with hereditary angioedema

Hereditary angioedema (HAE) is a rare genetic disorder characterized by severe, recurrent and unpredictable swelling in various organs and tissues of the body that can be painful, debilitating and life-threatening. A new study presented at this year's annual scientific meeting of the American College of Allergy, Asthma and Immunology (ACAAI) in Louisville, Kentucky, shows that treatment using CRISPR genome editing technology successfully relieved swelling and reduced the frequency of attacks.

NTLA-2002 is a first-of-its-kind, systemically delivered CRISPR genome editing candidate being developed for HAE. It is designed to turn off the KLKB1 gene in liver cells, thereby reducing the production of a specific protein called kallikrein, whose uncontrolled activity is responsible for the recurrent, debilitating and potentially fatal swelling attacks that occur in people with HAE.”

Hilary Longhurst, MD, PhD, clinical immunologist, lead author of the study

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Preliminary clinical data from the Phase 1 first-in-human study of NTLA-2002 will be presented at this year's ACAAI conference specifically for patients with HAE (both men and women) previously treated with escalating doses of 25 mg, 50 mg and 75 mg.

“While data are still pending, all patients treated with a single dose of NTLA-2002 in the 25 and 75 mg cohorts demonstrated rapid and significant reductions in plasma kallikrein levels,” says Dr. Danny Cohn, senior author of the study. “Of the three patients in the 25 mg dose cohort who reached a predetermined time point at which their HAE attacks were assessed, all patients also had a significant reduction in HAE attacks.”

At both the 25 and 75 mg doses, NTLA-2002 was generally well tolerated and most adverse events were mild in severity. The most common adverse reactions were infusion-related reactions, which were mostly grade 1 and resolved within one day. To date, no dose-limiting toxicities, no serious adverse events, and no grade 3 or higher adverse events have been observed.

“These early data support NTLA-2002 as a potential one-time treatment to cure HAE symptoms,” says Dr. Longhurst. “Clinical development continues for this program, with the randomized Phase 2 portion of the clinical trial scheduled to begin in the first half of 2023.”

Source:

American College of Allergy, Asthma and Immunology (ACAAI)

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