NGGT002 / NGGT (Suzhou) Biotech - LARVOL DELTA

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