PR1100

PR1100 (also known as AX-0810) is an investigational RNA editing oligonucleotide developed by ProQR Therapeutics for the treatment of hereditary angioedema (HAE). Utilizing the proprietary Axiomer platform, PR1100 is designed to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to the Kallikrein B1 (KLKB1) mRNA. The drug facilitates a site-specific adenosine-to-inosine (A-to-I) edit, which the cellular translation machinery interprets as a guanosine (G). This editing event is intended to introduce a missense mutation or a premature stop codon that results in the knockdown of prekallikrein protein production. By lowering systemic prekallikrein levels, PR1100 aims to inhibit the kallikrein-kinin pathway and prevent the excessive formation of bradykinin, the primary mediator of the painful and potentially life-threatening swelling episodes characteristic of HAE. PR1100 is currently being evaluated in Phase 1/2 clinical trials.