Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema | Zendy

Danny M. Cohn | Zendy; Nicholas J. Viney | Zendy; Lauré M. Fijen | Zendy; Eugene Schneider | Zendy; Veronica J. Alexander | Zendy; Shuting Xia | Zendy; Gwendolyn E. Kaeser | Zendy; Charvi Nanavati | Zendy; Brenda F. Baker | Zendy; Richard S. Geary | Zendy; Marcel Levi | Zendy; Joost C.M. Meijers | Zendy; Erik S.G. Stroes | Zendy

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Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema

Author(s) -

Danny M. Cohn,

Nicholas J. Viney,

Lauré M. Fijen,

Eugene Schneider,

Veronica J. Alexander,

Shuting Xia,

Gwendolyn E. Kaeser,

Charvi Nanavati,

Brenda F. Baker,

Richard S. Geary,

Marcel Levi,

Joost C.M. Meijers,

Erik S.G. Stroes

Publication year - 2020

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1915035

Subject(s) - hereditary angioedema , prekallikrein , medicine , genetics , kallikrein , biology , dermatology , biochemistry , enzyme

Hereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-L Rx is a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to hepatocytes. In a compassionate-use pilot study, two patients with severe bradykinin-mediated angioedema were initially administered weekly subcutaneous injections of the unconjugated parent drug, IONIS-PKK Rx , for 12 to 16 weeks, after which they received IONIS-PKK-L Rx at a dose of 80 mg every 3 to 4 weeks for 7 to 8 months. Treatment was accompanied by a reduction in the angioedema attack rate. (Funded by Amsterdam UMC.).

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