NGGT001 / Nuojiebei Biotech - LARVOL DELTA

Bietti crystalline corneoretinal dystrophy: Advances in understanding and gene therapeutic approaches. (PubMed, Mol Vis) - "We specifically analyze the clinical outcomes of three investigational gene therapy products-ZVS101e, NGGT001, and VGR-R01-focusing on their preliminary efficacy, safety profiles, and tolerability. Future optimization of these approaches should carefully consider administration sites and modalities, injection volumes, and disease severity at intervention. With gene replacement therapy for BCD advancing through late-stage clinical development, regulatory approval and clinical implementation may be anticipated in the near future."

Journal • Review • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects (clinicaltrials.gov) - P1/2 | N=12 | Active, not recruiting | Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. | Trial primary completion date: Mar 2025 ➔ Nov 2025

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial. (PubMed, JAMA Ophthalmol) - P1 | "To evaluate safety and vision outcomes following gene therapy with adeno-associated virus (AAV) encoding CYP4V2 (rAAV-hCYP4V2, NGGT001 [Next Generation Gene Therapeutics])...While improvement in BCVA was noted, the magnitude was within test-retest values typically noted in eyes with very low levels of visual acuity, and BCVA improvement in both the study and nonstudy eyes could be related to a learning effect, with greater improvement in the study eye possibly related to study eyes' being the worse-seeing eye. ClinicalTrials.gov Identifier: NCT06302608."

Clinical • Journal • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders

Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects (clinicaltrials.gov) - P1/2 | N=12 | Active, not recruiting | Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.

New P1/2 trial • Inherited Retinal Dystrophy • Ophthalmology

Clinical Periodic Study Report on Safety and Efficacy of NGGT001 for Treating Bietti's Crystalline Dystrophy (ASGCT 2024) - "These results of the IIT study demonstrated the safety of NGGT001 and highlighted that NGGT001 achieved preliminary efficacy in adult BCD patients. Plain Language Summary: of an early stage clinical study showed that NGGT001, an AAV gene therapy product was safe and effective in treating patients with Bietti's crystalline corneoretinal dystrophy."

Clinical • Gene Therapies • Inherited Retinal Dystrophy • Musculoskeletal Diseases • Orthopedics

Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy (clinicaltrials.gov) - P1 | N=6 | Active, not recruiting | Sponsor: Xiamen Ophthalmology Center Affiliated to Xiamen University

New P1 trial • Inherited Retinal Dystrophy • Ophthalmology