Marisa Wexler, MS, senior science writer —

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to NTLA-2002, an experimental gene-editing therapy that Intellia Therapeutics is developing to treat hereditary angioedema (HAE). RMAT designation is granted to therapies with the potential to treat, reverse, or cure serious or life-threatening…

The U.S. Food and Drug Administration (FDA) has given Intellia Therapeutics permission to open U.S. sites in its ongoing clinical trial of NTLA-2002, an investigational gene-editing therapy for hereditary angioedema (HAE). Specifics are not yet available on the number or location of U.S. sites for the global Phase…

Astria Therapeutics has initiated a Phase 1b/2 trial called ALPHA-STAR to test STAR-0215, the company’s investigational therapy to prevent swelling attacks in people with hereditary angioedema (HAE). ALPHA-STAR (NCT05695248) intends to enroll 18 adults with HAE types 1 or 2 who have had at least four swelling…

The first group of participants has been dosed in a Phase 1 clinical trial evaluating the safety and pharmacological properties of ADX-324, an experimental treatment for hereditary angioedema (HAE) that is being developed by ADARx Pharmaceuticals. “Dosing of our first participants in this trial is a major milestone…

NTLA-2002, an experimental gene-editing therapy for hereditary angioedema (HAE) being developed by Intellia Therapeutics, has been awarded an innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), the company announced. This designation is the first step in the U.K.’s Innovative Licensing and Access…

Most hereditary angioedema (HAE) patients in a Phase 1/2 clinical trial have gone months without a swelling attack following a single infusion of the experimental gene-editing therapy NTLA-2002. The remaining patients haven’t yet reached the pre-specified 16-week follow-up period for their attack rates to be analyzed, but their comparable…

People with hereditary angioedema (HAE) who switched to Orladeyo (berotralstat) from other preventive treatments generally experience a reduction in swelling attacks. That is according to real-world data presented by BioCryst Pharmaceuticals, Orladeyo’s developer, during the 2022 Annual Scientific Meeting of the American College of Allergy, Asthma…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Takeda to extend the approval of Takhzyro (lanadelumab) for children as young as 2 with hereditary angioedema (HAE). The application has been given priority review status and a decision from is expected in…

Japan’s Ministry of Health, Labour and Welfare has approved Berinert S.C. Injection 2000 — a C1-inhibitor replacement therapy designed to be self-administered at home via injection under the skin twice per week — for preventing swelling attacks in people with hereditary angioedema (HAE). The therapy has been approved in…

The European Patent Office has granted patent protection to Verseon covering the company’s experimental plasma kallikrein inhibitors (PKIs). Two of these investigational medications, called VE-4666 and VE-4062, are currently in preclinical development as potential treatments of hereditary angioedema (HAE). “We’re very pleased with the European Patent Office’s…