Saturday, July 11, 2026

pharma · Phenylketonuria · Achondroplasia

BioMarin Pharmaceutical Australia

BioMarin Pharmaceutical Australia is a pharma organization headquartered in San Rafael, USA. Primary therapeutic focus areas include Phenylketonuria, Achondroplasia, Duchenne Muscular Dystrophy, Phenylketonuria (PKU), Hy

770 Lindaro Street, San Rafael, CA 94901, US HQ
1997 Founded
3,171 Employees
TGA registrant Type
Company details
Status
Public
HQ
770 Lindaro Street, San Rafael, CA 94901, US
Founded
1997
Employees
3,171
Programs
91
Drugs
36
Patents
175
Clinical program

Dose 1 of BMN 331

Phase 2 · gene therapy · Hereditary Angioedema

Dose 1 of BMN 331 for Hereditary Angioedema is a Phase 2 program (gene_therapy) at BioMarin Pharmaceutical Australia Pty Ltd with 1 ClinicalTrials.gov record(s).

Internal code 331-201

Full intelligence profile for this program is being prepared.

At a glance

Sponsor
BioMarin Pharmaceutical Australia Pty Ltd
Phase
Phase 2
Modality
gene_therapy
Indication
Hereditary Angioedema
Status
active
Trials
1

Clinical trials