The Future of HAE Care: Understanding Gene Editing

Hereditary angioedema is a rare genetic condition. It is often called HAE. People with HAE experience severe swelling in different parts of the body. These swelling attacks can be painful and dangerous. For many years, treatment focused only on managing these symptoms. This required frequent shots or pills to prevent attacks.¹

Today, new research is changing the goal of care. Scientists are working on a way to fix the source of the problem. This new method is called gene editing. It may offer a future where people are free from both attacks and daily treatments.^1-3

Using “molecular scissors” to fix HAE

To understand gene editing, think of your DNA as an instruction manual for your body. In HAE, there is a specific page in that manual that causes trouble. This page belongs to a gene called KLKB1. This gene tells your body to make a protein called kallikrein. Too much kallikrein leads to the swelling seen in HAE.¹

Scientists have created a tool called CRISPR-Cas9. You can think of this tool as "molecular scissors.” These scissors are very precise. They are designed to find the KLKB1 gene and "turn off" the gene. This stops the body from making too much kallikrein and bradykinin. By fixing the source, the body no longer triggers the swelling process.^1,2

A single dose for long-term health

The way people receive this treatment is different from current drugs. Most HAE drugs require shots every 2 weeks or pills every day. Gene editing is designed to be a one-time treatment. It is given as a single liquid dose into a vein. This is called an intravenous infusion or IV infusion.^1,2

The infusion usually takes place in an outpatient clinic. A person sits in a chair while the treatment slowly enters their bloodstream. This single dose travels to the liver to start the gene-editing process. The goal is for this 1 session to provide health benefits that last for years. This could remove the need for constant medical treatment.^1,2

Success in clinical trials

Treatment options have come a long way in recent years, with new drug approvals and potential new treatments coming soon. The Phase 3 HAELO clinical trial has shown very positive results for a drug called lonvoguran ziclumeran. It is also known as lonvo-z (formerly called NTLA-2002). This clinical trial included adults and teenagers with HAE.^2,3

The results were impressive. A single dose of the drug reduced monthly swelling attacks by 87 percent compared to a placebo. In this group, 62 percent of people were entirely free from attacks for 6 months. These people also did not need any other HAE treatments during that time.²

Safety and side effects

Safety is a top priority in these trials. Most side effects reported by people in the study were mild or moderate. The most common issues included:^1,2

• Reactions at the infusion site
• Headache
• Feeling tired or fatigued

There were no serious safety concerns or major liver problems reported in the Phase 3 study. These results give doctors confidence that the treatment is well-tolerated.^1,2

The road to approval

The path to making this treatment available to those with HAE is moving quickly. In April 2026, the company making the drug started the official application process with the US Food and Drug Administration (FDA). This is called a Biologics License Application (BLA).²

The FDA is expected to review the data throughout the rest of 2026. If the FDA approves the drug, it could be available for people with HAE in the first half of 2027.^2,3

Hope for the HAE community

Gene editing represents a major shift in how we think about healthcare for HAE. It moves beyond just stopping a current attack. It aims to prevent future attacks by changing the genetic cause. For people living with HAE, this could mean a life without the constant fear of swelling. While the process is still ongoing, the data shows that a one-time fix may soon be a reality.³