24 January 2023 - - US-based biotechnology company ADARx Pharmaceuticals, Inc has dosed the first cohort in a Phase 1 clinical study of ADX-324 for the treatment of hereditary angioedema, the company said.

HAE is a rare genetic disease that causes painful, rapid, and potentially life-threatening swelling attacks.

ADX-324 is a short-interfering RNA (siRNA) designed to reduce the production of PKK, a protein critical to the etiology of HAE.

The study is being conducted in Australia as a randomized, placebo-controlled, double-blind, single ascending dose trial in healthy volunteers with an expansion cohort in patients with HAE.

The primary objective of the study is to evaluate the safety and tolerability of ADX-324 when administered by subcutaneous injection.

ADARx Pharmaceuticals, Inc., located in San Diego, is a clinical stage biotechnology company committed to turning cutting-edge science into life-saving therapeutics.

ADARx is developing a proprietary RNA targeting platform, including oligonucleotides for inhibition, degradation, and editing, together with novel oligonucleotide delivery technologies including its PLR platform and SPE technology.

ADARx has a pipeline of RNA targeting therapeutics for treating diseases across a range of therapeutic areas including genetic, cardiometabolic, complement-mediated and central nervous system diseases.