A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

NCTID	NCT05121376 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Inactive
Indication	Hereditary Angioedema
Disease Ontology Term	DOID:14735
Compound Name	BMN 331
Sponsor	BioMarin Pharmaceutical
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	44
Results Posted	Not Available

Target Gene/Variant	SERPING1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Liver
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type
Dose 1	6E13 vg/kg
Dose 2	Other unknown doses
Dose 3
Dose 4
Dose 5

No.of Trial Sites	16
Locations	United States,Australia,Spain

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Biomarin announced they were discontinuing this program April 2024