Hereditary Angioedema Clinical Trials

HAE is a rare genetic condition characterized by spontaneous swelling that can be life-threatening by blocking airways and preventing breathing. The main type of HAE is caused by mutations in the SERPING1 gene. This gene encodes a protein called C1-INH which plays an important role in controlling certain types of swelling in the body. When C1-INH is missing or not working correctly, sudden, unexpected swelling events can occur.

Learn more about our hereditary angioedema clinical trials below.

Study 331-201 Recruiting

Study 331-201: A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Subjects with Hereditary Angioedema (HAE) due to Human C1 Esterase Inhibitor (C1-INH) Deficiency

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Indication

Hereditary Angioedema (HAE)

Goal

This open label, two-part study aims to establish the safety, tolerability, and efficacy of BMN 331

Investigational therapeutic

BMN 331

Status

This study is currently recruiting participants

Study Type

Phase 1/2

Study Website

https://haegenestudy.com/