Donidalorsen

Donidalorsen (API)

Research Application:
Donidalorsen API is an antisense oligonucleotide (ASO) under investigation for the treatment of hereditary angioedema (HAE) and related inflammatory conditions. It is studied in the context of RNA-targeted therapies, aiming to reduce the expression of plasma prekallikrein (KLKB1 mRNA). Researchers use Donidalorsen to explore gene silencing mechanisms, dose-dependent pharmacokinetics, and long-term control of bradykinin-mediated inflammation.

Function:
Donidalorsen functions by selectively binding to KLKB1 mRNA, reducing the production of plasma prekallikrein — a key enzyme in the kallikrein-kinin system responsible for triggering swelling and inflammation in HAE. By lowering kallikrein levels, Donidalorsen helps prevent HAE attacks and reduces disease burden. As an API, it serves as the core therapeutic component in the development of long-acting, subcutaneously administered treatments for HAE.