Hereditary Angioedema News and Research

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Phase 3 study of in vivo CRISPR therapy for hereditary angioedema successfully completed

Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first-ever Phase 3 study of an in vivo CRISPR therapy.

13 Jun 2026

Studies reveal how hereditary angioedema disrupts the lives of children and families

Two new studies being presented at the 2025 American College of Allergy, Asthma and Immunology (ACAAI) Annual Scientific Meeting in Orlando shed light on how hereditary angioedema (HAE) – a rare, potentially life-threatening disease which causes unpredictable swelling – profoundly affects the daily lives, emotional wellbeing, and medical experiences of young patients and their caregivers.

6 Nov 2025

Single treatment with CRISPR gene editing therapy offers hope for hereditary angioedema patients

A single treatment with, a CRISPR-Cas9 based gene editing therapy, is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful and sudden onset of swelling, sometimes resulting in death.

25 Oct 2024

Berotralstat: A promising oral treatment for hereditary angioedema with normal C1-inhibitor

BioCryst Pharmaceuticals, Inc. today announced new real-world evidence showing that patients with hereditary angioedema (HAE) who have normal C1-inhibitor (HAE-nC1-INH) level and function had a reduction in monthly attack rates after starting oral, once-daily berotralstat.

8 Jun 2024

Breakthrough gene-editing therapy transforms lives of patients with a hereditary disorder

A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher.

2 Feb 2024

Treatment using CRISPR genome editing alleviates swelling attacks in hereditary angioedema patients

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring, and unpredictable swelling attacks in various organs and tissues of the body, which can be painful, debilitating, and life-threatening.

10 Nov 2022

Study elucidates why it takes a long time to diagnose life-threatening rare diseases

Regular episodes of severe pains in the abdomen and mysterious swellings had become the new normal for Yuki - for 20 years.

19 Apr 2022

Latest ATS/CSL Behring research grant recipient focuses on COVID-19 ARDS study

The ATS Research Program is pleased to announce that William Zhang, MD, of Weill Cornell Medical Center is the recipient of the 2020-2021 ATS/CSL Behring Research Award in Acute Respiratory Distress Syndrome.

13 Jul 2021

Innate immune system overreacts in patients with severe COVID-19

In patients with severe COVID-19, the innate immune system overreacts. This overreaction may underlie the formation of blood clots (thrombi) and deterioration in oxygen saturation that affect the patients.

23 Feb 2021

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

COVID-19 patients with hypoxia respond positively to a treatment, study shows

Covid-19 patients with hypoxia respond positively to icatibant treatment, Radboud university medical center researchers wrote in JAMA Network Open.

19 Aug 2020

Computational analyses suggest new pathway for inflammatory response to SARS-CoV-2

Analyses of lung fluid cells from COVID-19 patients conducted on the nation's fastest supercomputer point to gene expression patterns that may explain the runaway symptoms produced by the body's response to SARS-CoV-2.

28 Jul 2020

C-Path's Transplant Therapeutics Consortium receives positive FDA response for its Letter of Intent

Critical Path Institute announced today that its Transplant Therapeutics Consortium has received a positive response to its Letter of Intent from the U.S. Food and Drug Administration detailing the decision to accept the Composite Biomarker Panel (iBox Scoring System) into the Center for Drug Evaluation and Research (CDER) Biomarker Qualification Program.

17 Jun 2020

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

Seattle Children's Research Institute, one of the top pediatric research institutions in the world, and global biotechnology leader CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases.

2 Jun 2020

Radboudumc researchers find essential mechanism in the disease process of COVID-19

Researchers at the Radboud University Medical Center seem to have found an essential mechanism in the disease process of COVID-19, which has so far been overlooked.

29 Apr 2020

New, previously unknown form of familial cold urticaria discovered

Skin rash combined with head and joint pain: these are the symptoms which patients with familial (hereditary) cold urticaria develop when exposed to temperatures below 15 °C.

27 Apr 2020

Treatments for rare diseases affecting children are increasing, study suggests

The number of treatments for rare diseases affecting children has increased, a new study suggests. But federal incentives intended to encourage drug development for rare conditions are being used more often to expand the use of existing drugs rather than for creating new ones.

3 Mar 2020

Study provides new understanding of mysterious 'hereditary swelling'

An upper arm that swells to twice its normal size. A stomach that inexplicably swells up causing colic pain, vomiting or diarrhea. Or a life-threatening variant with severe swelling of the face, lips, tongue and larynx, bringing the risk of obstructed airways and death by suffocation.

11 Dec 2018

New antibody can reduce swelling attacks in patients with hereditary angioedema

Patients with hereditary angioedema experience recurrent and sometimes life-threatening swelling attacks. Researchers from Charité - Universitätsmedizin Berlin have tested an antibody capable of reducing the frequency of these swelling attacks by more than 90 percent.

28 Nov 2018

Hereditary Angioedema News and Research

Phase 3 study of in vivo CRISPR therapy for hereditary angioedema successfully completed

Studies reveal how hereditary angioedema disrupts the lives of children and families

Single treatment with CRISPR gene editing therapy offers hope for hereditary angioedema patients

Berotralstat: A promising oral treatment for hereditary angioedema with normal C1-inhibitor

Breakthrough gene-editing therapy transforms lives of patients with a hereditary disorder

Treatment using CRISPR genome editing alleviates swelling attacks in hereditary angioedema patients

Study elucidates why it takes a long time to diagnose life-threatening rare diseases

Latest ATS/CSL Behring research grant recipient focuses on COVID-19 ARDS study

Innate immune system overreacts in patients with severe COVID-19

Providing personalized care for bleeding disorders

Providing personalized care for bleeding disorders

COVID-19 patients with hypoxia respond positively to a treatment, study shows

Computational analyses suggest new pathway for inflammatory response to SARS-CoV-2

C-Path's Transplant Therapeutics Consortium receives positive FDA response for its Letter of Intent

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

Radboudumc researchers find essential mechanism in the disease process of COVID-19

New, previously unknown form of familial cold urticaria discovered

Treatments for rare diseases affecting children are increasing, study suggests

Study provides new understanding of mysterious 'hereditary swelling'

New antibody can reduce swelling attacks in patients with hereditary angioedema

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