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Hereditary Angioedema clinical trials at UCSD

1 research study open to eligible people

Showing trials for
  • Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

    open to eligible people ages 18 years and up

    This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency. The pharmaceutical form of BMN 331 is a solution for intravenous infusion.

    San Diego, California and other locations

Our lead scientists for Hereditary Angioedema research studies include .

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