domofenogene zalfaparvovec (BMN 307) / BioMarin - LARVOL DELTA

AAV Gene Therapy Study for Subjects With PKU (clinicaltrials.gov) - P1/2; N=100; Active, not recruiting; Sponsor: BioMarin Pharmaceutical; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

AAV Gene Therapy Study for Subjects With PKU (clinicaltrials.gov) - P1/2; N=100; Recruiting; Sponsor: BioMarin Pharmaceutical; Trial completion date: Dec 2026 ➔ Dec 2027; Trial primary completion date: Dec 2026 ➔ Dec 2027

Clinical • Trial completion date • Trial primary completion date • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

AAV Gene Therapy Study for Subjects With PKU (clinicaltrials.gov) - P1/2; N=100; Recruiting; Sponsor: BioMarin Pharmaceutical

Clinical • New P1/2 trial • Metabolic Disorders • Phenylketonuria • Rare Diseases