NGGT002 / NGGT (Suzhou) Biotech - LARVOL DELTA

Safety and Efficacy Study of NGGT002 in Adult Patients With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. | Trial completion date: Jul 2031 ➔ Jun 2030 | Trial primary completion date: Jul 2026 ➔ Aug 2027

Trial completion date • Trial primary completion date • Metabolic Disorders • Phenylketonuria • Rare Diseases

Safety and Efficacy Study of NGGT002 in PKU Adult Subjects (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: The First Affiliated Hospital of Bengbu Medical University | Recruiting ➔ Active, not recruiting

Enrollment closed • Metabolic Disorders • Phenylketonuria • Rare Diseases

Efficacy and safety of NGGT002 at week 52 and beyond in adults with Phenylketonuria (ASGCT 2026) - "Conclusion This study demonstrated that a single dose of NGGT002 was well-tolerated and effective in stably normalizing blood Phe levels in adult PKU patients. AAV gene therapy represents an encouraging approach for treating PKU."

Clinical • First-in-human • Gene Therapies • Genetic Disorders • Metabolic Disorders • Phenylketonuria • Rare Diseases

Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.

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

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: NGGT INC. | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

AAV Gene Therapy Clinical Study in Adult Classic PKU (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: NGGT INC. | Initiation date: May 2024 ➔ Nov 2024

Gene therapy • Trial initiation date • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

Safety and Efficacy of NGGT002 (rAAV8-hPAH) in Adults with Phenylketonuria (PKU): Results from an Investigator Initiated Study (ASGCT 2024) - P1 | "A single infusion of NGGT002 gene therapy is well tolerated in adults with PKU. Encouraging evidence of significant and stable efficacy has been observed. These results support further clinical development of NGGT002."

Clinical • Gene Therapies • Genetic Disorders • Hepatology • Liver Failure • Metabolic Disorders • Phenylketonuria • Psychiatry • Rare Diseases

AAV Gene Therapy Clinical Study in Adult Classic PKU (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.

Gene therapy • New P1/2 trial • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

AAV8 Gene Therapy in PKU Adult Subjects (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: First Affiliated Hospital Bengbu Medical College

Gene therapy • New P1 trial • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases