Marisa Wexler, MS, senior science writer —

Ionis Pharmaceuticals has finished enrollment in a Phase 3 clinical trial testing the ability of its experimental therapy donidalorsen to prevent swelling attacks in people with hereditary angioedema (HAE) types 1 or 2. Results from the placebo-controlled study, dubbed OASIS-HAE (NCT05139810), are expected in the first half…

An experimental gene-editing therapy for hereditary angioedema (HAE), called NTLA-2002, has been administered to the first participant in the Phase 2 portion of a clinical trial, according to its developer, Intellia Therapeutics. “We are pleased to announce dosing has begun in the Phase 2 study of NTLA-2002,”…

A pregnant woman with type 3 hereditary angioedema (HAE) had potential swelling attacks effectively controlled during both pregnancy and breastfeeding with the use of subcutaneous or under-the-skin injections of a plasma-derived C1 esterase inhibitor (C1-INH), according to a case report from Spain. Researchers say this may be the first…

A woman with colon cancer developed angioedema as a side effect of treatment with an immune checkpoint inhibitor (ICI), a recently developed type of cancer immunotherapy. Researchers advised that “clinicians, pharmacists, and patients should be aware of this rare side effect of ICIs,” adding that facial angioedema can be…

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…