New Treatments on the Horizon

Adverum – ADVM-053: A gene therapy candidate being investigated as a potential single‑administration treatment to provide sustained levels of the C1 esterase inhibitor protein. Learn more here.

Alnylam Pharmaceuticals – AKN-F12: A RNA interference (RNAi) drug to knockdown factor XII. Learn more here.

Arrowhead – ARC-F12: An RNAi therapeutic inhibiting the production of Factor XII. Learn more here.

Attune Pharmaceuticals – ATN-249: An orally-administered plasma kallikrein inhibitor. Learn more here.

BioCryst – BCX7353: A once-daily oral treatment (capsule or liquid) inhibiting plasma kallikrein. Learn more here and find information on the clinical trial in Canada.

BioMarin Pharmaceutical Inc. – BMN 331: An investigational gene therapy intended to provide C1 esterase inhibitor to treat hereditary angioedema.

CSL Behring – CSL312: A humanised anti-factor XIIa monoclonal antibody for subcutaneous treatment. Learn more here and find information for the clinical trial in Canada.

Ionis – IONIS-PKK-L_Rx: A Generation 2⁺ ligand-conjugated antisense drug designed to reduce prekallikrein production. Learn more here.

Takeda Pharmaceuticals – TAK-743: Lanadelumab (Takhzyro), a fully human monoclonal antibody inhibitor of plasma kallikrein for the prevention of Hereditary Angioedema (HAE) Attacks in pediatric subjects 2-12 years of age Learn more here and find information on the clinical trial in Canada.