Hereditary angioedema: alleviation by gene editing

Patients with hereditary angioedema suffer from severe swelling of various body tissues. Scientists have now introduced a new therapy: Using the CRISPR method, a protein that triggers seizures can be inhibited.

Hereditary angioedema (HAE) is a rare, genetic condition characterized by severe, recurrent swelling in various organs and tissues. These are not only painful, but can also be life-threatening. A recent study has now shown that treatment using CRISPR genome editing technology can reduce swelling and reduce the frequency of seizures.

promising candidate

The research team has now presented preliminary clinical data from the first phase 1 study with a promising candidate: NTLA-2002. “NTLA-2002 is a unique, system-directed CRISPR genome editing candidate being developed for HAE,” explains lead author Dr. Hilary Longhurst.

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“It is thought to turn off the KLKB1 gene in liver cells, thereby reducing the production of a particular protein called kallikrein, whose uncontrolled activity is responsible for the recurring, debilitating and potentially fatal bouts of swelling that people with HAE experience.” Longhurst and her team treated plus patients with HAE who received treatment with escalating doses of 25 mg, 50 mg, and 75 mg.

A significant effect

“Although data are not yet available, we observed a rapid and significant reduction in plasma kallikrein levels in all patients treated with a single dose of NTLA-2002 in the 25 and 75 mg dose cohorts,” said study author Dr. Danny Cohn. “During the period in which the HAE seizures of patients in the 25 mg cohort were analyzed, a significant decrease in seizures was also observed in all patients.”

Well tolerated – few side effects

Both the 25mg and 75mg doses of NTLA-2002 are well tolerated, with most side effects — such as infusion-related reactions — mild, according to the researchers. No dose-limiting toxicities or other serious side effects have been observed to date. “These early data support NTLA-2002 as a potential one-time treatment to cure the symptoms of HAE,” said Dr. longhurst The second part of clinical development is scheduled to begin in the first half of 2023.

This text is based on a press release from the American College of Allergy, Asthma, and Immunology. We have an original publication for you here and referenced in the text.

Image credit: Miles Burke, unsplash.

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